F10 modulators(Coagulation factor X modulators), factor IX modulators(Coagulation factor IX modulators), factor VII modulators(Coagulation factor VII modulators)

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication

Hemophilia AHemophilia B

Inactive Indication-

Originator Organization

Baxalta US, Inc.

Active Organization

Universidade Federal De Sao PauloTakeda Pharmaceuticals U.S.A., Inc.

Baxter Healthcare Ltd

+ [1]

Inactive Organization

Baxalta Innovations GmbH

Baxalta, Inc.

Drug Highest PhaseApproved

First Approval Date

US (21 Dec 1979),

Hemophilia AHemophilia B

Regulation-

Clinical Trials associated with Anti-Inhibitor Coagulant Complex (Baxalta US)

NCT04563520 / RecruitingPhase 3IIT

aPCC and Emicizumab Safety Study in Congenital Hemophilia A Patients With Inhibitors (SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis)

The purpose of the aPCC-emicizumab safety study is to prospectively investigate the safety and hemostatic efficacy of a personalized dose of aPCC in children and adults with hemophilia A and inhibitors on emicizumab prophylaxis during acute bleeding events or prior to procedures.

Start Date01 Feb 2025

Sponsor / Collaborator

Emory University

[+1]

NCT05020483 / WithdrawnPhase 2IIT

Activated Prothrombin Complex Concentrate FEIBA to Optimize Postcardiopulomonary Bypass Hemostasis in Pediatric Cardiac Patients

The purpose of this research is to understand if the medication called FEIBA helps to stop bleeding and decrease transfusion of blood products to small children operated on the heart.

Start Date01 Oct 2021

Sponsor / Collaborator

Mayo Clinic

NCT04205175 / Active, not recruitingPhase 4IIT

A Single-center, Open-Label, Dose Escalation Study Evaluating the Safety of in Vivo Administration of FEIBA in Congenital Hemophilia A Patients With Inhibitors on Emicizumab

Hemophilia A is a severe, life-long, genetic bleeding disorder characterized by a deficiency of factor VIII (FVIII), a crucial cofactor of the coagulation system. The mainstay of hemophilia treatment is factor replacement therapy with FVIII clotting factor concentrates (CFC) and these can be given episodically in response to bleeding or prophylactically to prevent bleeding. The main adverse effect of FVIII CFC is the development of neutralizing anti-drug antibodies termed inhibitors, and these render replacement therapy less effective if they are low titer inhibitors or completely ineffective if they are of the high titer variety. These so-called 'inhibitor patients' cannot rely on FVIII CFC for their treatment and are treated with other CFC called bypassing agents such as activated prothrombin complex concentrate (aPCC/Feiba). While these agents can be effective in some patients for prophylaxis, they are not as effective for bleed prevention as FVIII CFC for patients without inhibitors.Recently, emicizumab (Hemlibra, Roche), was developed and licensed for the prevention of bleeding in patients with hemophilia A with and without inhibitors. However, patients in the clinical trials for emicizumab have developed thrombotic adverse events and only patients who received doses of Feiba of >100 IU/kg/24 hours for more than 24 hours developed thrombosis.
As a result of the above data, recommendations have been to either avoid altogether in patients on emicizumab, or to be very cautious about using it to treat breakthrough bleeding. With this in mind, we propose to study the in vivo combination of Feiba in patients with inhibitors on emicizumab.

Start Date01 Jul 2020

Sponsor / Collaborator

Children's Hospital Los Angeles

[+1]

100 Clinical Results associated with Anti-Inhibitor Coagulant Complex (Baxalta US)

100 Translational Medicine associated with Anti-Inhibitor Coagulant Complex (Baxalta US)

100 Patents (Medical) associated with Anti-Inhibitor Coagulant Complex (Baxalta US)

330

Literatures (Medical) associated with Anti-Inhibitor Coagulant Complex (Baxalta US)

01 Dec 2024·ANESTHESIOLOGY

Factor Eight Inhibitor Bypass Activity Use in Cardiac Surgery: A Propensity-matched Analysis of Safety Outcomes

Article

Author: Chang, Alan L. ; Jackson, Ethan ; Wirtz, Katherine ; Panigrahi, Anil K. ; Aghaeepour, Nima ; Parsons, Cody ; Nielson, Elaina ; Nicholas, Joshua A. ; Chakraborty, Dipro ; Harrison, Natasha

Background:

Bleeding during cardiac surgery may be refractory to standard interventions. Off-label use of factor eight inhibitor bypass activity (FEIBA) has been described to treat such bleeding. However, reports of safety, particularly thromboembolic outcomes, show mixed results, and reported cohorts have been small.

Methods:

Adult patients undergoing cardiac surgery on cardiopulmonary bypass between July 1, 2018, and June 30, 2023, at Stanford Hospital (Stanford, California) were reviewed (n = 3,335). Patients who received FEIBA to treat postcardiopulmonary bypass bleeding were matched with those who did not by propensity scores in a 1:1 ratio using nearest neighbor matching (n = 352 per group). The primary outcome was a composite outcome of thromboembolic complications including any one of deep vein thrombosis, pulmonary embolism, unplanned coronary artery intervention, ischemic stroke, and acute limb ischemia, in the postoperative period. Secondary outcomes included renal failure, reoperation, postoperative transfusion, intensive care unit length of stay, and 30-day mortality.

Results:

A total of 704 encounters was included in this propensity-matched analysis. The mean dose of FEIBA administered was 7.3 ± 5.5 U/kg. In propensity-matched multivariate logistic regression models, there was no statistically significant difference in odds ratios for thromboembolic outcomes, intensive care unit length of stay, or mortality. Patients who received more than 750 U FEIBA had an increased odds ratio for acute renal failure (odds ratio, 4.14; 95% CI, 1.61 to 10.36; P < 0.001). In multivariate linear regression, patients receiving FEIBA were transfused more plasma and cryoprecipitate postoperatively. However, only the dose range of 501 to 750 U was associated with an increase in transfusion of erythrocytes (β, 2.73; 95% CI, 0.68 to 4.78; P = 0.009) and platelets (β, 1.74; 95% CI, 0.85 to 2.63; P < 0.001).

Conclusions:

Low-dose FEIBA administration during cardiac surgery does not increase risk of thromboembolic events, intensive care unit length of stay, or mortality in a propensity-matched cohort. Higher doses were associated with increased acute renal failure and postoperative transfusion. Further studies are required to establish the efficacy of activated factor concentrates to treat refractory bleeding during cardiac surgery.

Editor’s Perspective:

What We Already Know about This Topic:

What This Article Tells Us That Is New:

01 Sep 2024·JOURNAL OF CARDIOTHORACIC AND VASCULAR ANESTHESIA

Factor Eight Inhibitor Bypassing Activity as First-line Therapy for Coagulopathy in Cardiac Surgery

Article

Author: Kim, Hyungjoo ; Hartman, Alan ; Huang, Xueqi ; Yu, Pey-Jen ; Manetta, Frank

OBJECTIVES:

To compare the outcomes of factor eight inhibitor bypassing activity (FEIBA) versus fresh frozen plasma (FFP) as the primary treatment for postoperative coagulopathy in patients undergoing cardiac surgery.

DESIGN:

A retrospective, propensity-matched study.

SETTING:

A single, tertiary hospital.

PARTICIPANTS:

Patients who underwent noncoronary cardiac surgery with cardiopulmonary bypass between 2015 and 2023.

INTERVENTIONS:

None.

MEASUREMENTS AND MAIN RESULTS:

We stratified patients into 2 groups based on whether they received intraoperative FFP or FEIBA; cases using both were excluded. We analyzed 434 cases, with 197 receiving FFP and 237 receiving FEIBA. After propensity matching, there was no significant difference in the proportion of the patients who required packed red blood cell transfusions (p = 0.08). However, of those who required packed red blood cell transfusions, patients in the FEIBA group required significantly fewer units of packed red blood cells (p < 0.001). Significantly fewer patients in the FEIBA group required platelet (p < 0.001) and cryoprecipitate (p < 0.001) transfusions. The FEIBA group showed decreased prolonged postoperative intubation (p = 0.05), decreased intensive care unit length of stay (p = 0.04), and lower 30-day readmission rates (p = 0.03). There were no differences in the rates of thrombotic complications between the 2 cohorts.

CONCLUSIONS:

In the initial treatment of postcardiopulmonary bypass coagulopathy, FEIBA may be more effective than FFP in decreasing blood product transfusions and readmission rates. Further studies are needed to explore the potential routine use of FEIBA as first-line agent in this patient population.

01 Jun 2024·Journal of thrombosis and haemostasis : JTH

Emicizumab promotes factor Xa generation on endothelial cells

Article

Author: Ellsworth, Patrick ; Key, Nigel S ; Fager, Ammon M ; Monroe, Dougald M ; Hoffman, Maureane

BACKGROUND:

Until recently, the treatment of hemophilia A relied on factor (F)VIII replacement. However, up to one-third of patients with severe hemophilia A develop neutralizing alloantibodies that render replacement therapies ineffective. The development of emicizumab, a bispecific antibody that partially mimics FVIIIa, has revolutionized the treatment of these patients. However, the use of an activated prothrombin complex concentrate [FEIBA (Takeda)] to treat breakthrough bleeding in patients on emicizumab has been associated with thrombotic complications including a unique microangiopathy.

OBJECTIVES:

We hypothesized that the thrombotic complications observed with the combination of emicizumab and FEIBA might be due to excessive expression of procoagulant activity on the surface of endothelial cells.

METHODS:

We examined the ability of emicizumab to promote FX activation on endothelial cells using 2 cell culture models.

RESULTS:

We found that endothelial cells readily support emicizumab-mediated activation of FX by FIXa. The level of FXa generation depends on the concentration of available FIXa. The addition of FEIBA to emicizumab increased FXa generation in a dose-dependent manner on endothelial cells in both models. The rate of FXa generation was further enhanced by endothelial cell activation. However, unlike emicizumab, we found limited FXa generation in the presence of FVIII(a), which followed a significant lag time and was not dependent on FIXa concentration under these conditions.

CONCLUSION:

Emicizumab promotes FXa generation on the surface of endothelial cells, which is markedly enhanced in the presence of FEIBA. These findings demonstrate a potential mechanism for the thrombotic complications seen with the combined use of emicizumab and FEIBA.

News (Medical) associated with Anti-Inhibitor Coagulant Complex (Baxalta US)

05 Nov 2024

·www.businesswire.com

Genentech to Present New and Encouraging Long-term Follow-up Data Across Broad Hematology Portfolio at ASH 2024

SOUTH SAN FRANCISCO, Calif.--( BUSINESS WIRE )--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) announced today that it will present more than 40 abstracts across nine blood disorders at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 7-10, 2024 in San Diego, California. The data underscore Genentech’s commitment to advance patient outcomes in lymphoma with long-term follow-up of its approved medicines Polivy ® (polatuzumab vedotin-piiq), Lunsumio ® (mosunetuzumab-axgb) and Columvi ® (glofitamab-gxbm) as well as new investigational combination data. Key presentations include: Five-year data from the Phase III POLARIX study (abstract #469) reinforce the potential of Polivy in combination with Rituxan ® (rituximab), cyclophosphamide, doxorubicin and prednisone (R-CHP) to provide durable and lasting remissions and for the first time show a positive trend in overall survival (OS) for people with first-line diffuse large B-cell lymphoma (DLBCL), an area that previously had little advancement in nearly two decades. Extended follow-up data of up to four years from the pivotal GO29781 study of Lunsumio (abstract #4407) and NP30179 study of Columvi (abstract #865) show long-lasting remissions and immune system recovery after the end of treatment, supporting the use of fixed-duration bispecific antibodies for third-line or later (3L+) follicular lymphoma (FL) and DLBCL, respectively. First presentation of data for a subcutaneous formulation of Lunsumio monotherapy from the pivotal Phase II GO29781 study (abstract #1645) show high rates of deep and durable responses and low rates and severity of cytokine release syndrome in people with 3L+ FL. Subcutaneously administered Lunsumio could further improve the patient experience by combining shorter administration time with the existing benefits of a fixed-duration and outpatient therapy. New patient-reported outcomes data from the Phase III STARGLO study (abstract #5132) indicate comparable health-related quality of life between treatment arms, despite higher median number of cycles received with the Columvi combination (11 versus 4). Together with the significant improvement in OS observed in the study, these data support the potential benefit for patients with second-line or later DLBCL. New and updated data from investigational study combinations of Polivy with bispecific antibodies Lunsumio and Columvi in relapsed or refractory DLBCL, including the Phase Ib/II NP39488 (abstract #988) and Phase II GO40516 (abstract #989) studies, add to the growing body of evidence demonstrating the potential of novel bispecific antibody/Polivy combinations in earlier treatment lines, and support their ongoing Phase III development. Overview of key presentations featuring Genentech medicines Medicine Abstract title Abstract number/presentation details Polivy Five-Year Analysis of the POLARIX Study: Prolonged Follow-up Confirms Positive Impact of Polatuzumab Vedotin Plus Rituximab, Cyclophosphamide, Doxorubicin, and Prednisone (Pola-R-CHP) on Outcomes #469 oral presentation Session: 626. Aggressive Lymphomas: Clinical and Epidemiological: CARs, Bispecifics, and ADCs: Progress and Challenges in Aggressive B Cell Lymphoma Sunday, December 8, 2024 9:30 AM PST A Multicenter, Prospective, Observational Study of Pola-R-CHP in Patients With Previously Untreated Diffuse Large B-Cell Lymphoma (POLASTAR): A Preliminary Analysis #4475 poster presentation Session: 626. Aggressive Lymphomas: Clinical and Epidemiological: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST Lunsumio A Randomized Phase II Study of Mosunetuzumab SC Plus Polatuzumab Vedotin Demonstrates Improved Outcomes Versus Rituximab Plus Polatuzumab Vedotin in Patients (Pts) with Relapsed or Refractory (R/R) Large B-Cell Lymphoma (LBCL) #989 oral presentation Session: 627. Aggressive Lymphomas: Pharmacologic Therapies: Chemotherapy-free Combinations for Relapsed Aggressive Lymphomas Monday, December 9, 2024 5:30 PM PST Subcutaneous Mosunetuzumab Leads to High Rates of Durable Responses, Low Rates of Cytokine Release Syndrome, and Non-Inferior Exposure Compared with Intravenous Administration in Patients with Relapsed/Refractory Follicular Lymphoma: Primary Analysis of a Pivotal Phase II Study #1645 poster presentation Session: 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster I Saturday, December 7, 2024 5:30 - 7:30 PM PST Mosunetuzumab Continues to Demonstrate Clinically Meaningful Outcomes in Patients with Relapsed and/or Refractory Follicular Lymphoma after ≥2 Prior Therapies Including Those with a History of POD24: 4-Year Follow-up of a Pivotal Phase II Study #4407 poster presentation Session: 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST Mosunetuzumab Monotherapy Demonstrates Encouraging Activity and a Manageable Safety Profile in Patients with Heavily Pre-Treated Relapsed or Refractory Mantle Cell Lymphoma #1646 poster presentation Session: 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster I Saturday, December 7, 2024 5:30 - 7:30 PM PST Fixed-Duration Subcutaneous Mosunetuzumab is Active and has a Manageable Safety Profile in Patients with Previously Untreated, Low-Tumor Burden Follicular Lymphoma: Updated Results from the Phase II MorningSun Study #3008 poster presentation Session: 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster II Sunday, December 8, 2024 6:00 - 8:00 PM PST Travel Burden and Travel Costs of Bispecific Antibodies in Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma and Relapsed/Refractory Follicular Lymphoma #782 oral presentation Session: 902. Health Services and Quality Improvement: Lymphoid Malignancies: For a Better Tomorrow - Improving Access to Blood Cancer Treatments and Trials Monday, December 9, 2024 10:45 AM PST Columvi Fixed-duration Glofitamab Monotherapy Continues to Demonstrate Durable Responses in Patients with Relapsed or Refractory Large B-Cell Lymphoma: 3-year Follow-Up From a Pivotal Phase II Study #865 oral presentation Session: 627. Aggressive Lymphomas: Pharmacologic Therapies: New R-CHOP Combinations for Treatment Naïve DLBCL Sunday, December 8, 2024 Midday PST Glofitamab in Combination with Polatuzumab Vedotin Maintains Durable Responses and a Manageable Safety Profile in Patients with Heavily Pre-treated Relapsed/Refractory (R/R) Large B-Cell Lymphoma (LBCL) Including High-Grade B-Cell Lymphoma (HGBCL): Extended Follow-Up of a Phase Ib/II Study #988 oral presentation Session: 627. Aggressive Lymphomas: Pharmacologic Therapies: Chemotherapy-free Combinations for Relapsed Aggressive Lymphomas Monday, December 9, 2024 5:15 PM PST Glofitamab in Combination with Rituximab plus Ifosfamide, Carboplatin, and Etoposide shows Favorable Efficacy and Manageable Safety in Patients with Relapsed or Refractory Diffuse Large B-cell Lymphoma, Eligible for Stem Cell Transplant or Chimeric Antigen Receptor T-cell Therapy: Results from a Phase Ib Study #987 oral presentation Session: 627. Aggressive Lymphomas: Pharmacologic Therapies: Chemotherapy-free Combinations for Relapsed Aggressive Lymphomas Monday, December 9, 2024 5:00 PM PST Primary Results of Patient-Reported Outcomes in Patients with Relapsed/Refractory Diffuse Large B-cell Lymphoma Treated with Glofitamab plus Gemcitabine and Oxaliplatin (Glofit-GemOx) Versus Rituximab plus GemOx (R-GemOx) from the Phase III STARGLO Study #5132 poster presentation Session: 906. Outcomes Research: Lymphoid Malignancies Excluding Plasma Cell Disorders: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST Glofitamab Induces High Response Rates and Durable Remissions in Patients (Pts) with Heavily Pretreated Relapsed/Refractory (R/R) Mantle Cell Lymphoma (MCL), including those with a Poor Prognosis: Subgroup Results from a Phase I/II Study #1631 poster presentation Session: 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster I Saturday, December 7, 2024 5:30 - 7:30 PM PST A Healthcare Utilization Model Comparing Time Toxicity Between Glofitamab and Epcoritamab Treatment Regimens #3647 poster presentation Session: 902. Health Services and Quality Improvement: Lymphoid Malignancies: Poster II Sunday, December 8, 2024 6:00 - 8:00 PM PST Englumafusp alfa Englumafusp Alfa (CD19-4-1BBL) Combined with Glofitamab is Safe and Efficacious in Patients with R/R B-NHL: Extended Follow Up Analysis of the Dose-Escalation Part of Phase I Trial BP41072 #990 oral presentation Session: 627. Aggressive Lymphomas: Pharmacologic Therapies: Chemotherapy-free Combinations for Relapsed Aggressive Lymphomas Monday, December 9, 2024 5:45 PM PST Cevostamab Cevostamab in Patients with Heavily Pretreated Relapsed/Refractory Multiple Myeloma (RRMM): Updated Results from an Ongoing Phase I Study Demonstrate Clinically Meaningful Activity and Manageable Safety and Inform the Doses and Regimen for Combination Studies #1021 oral presentation Session: 654. Multiple Myeloma: Pharmacologic Therapies: Into the Future: New Drugs and Combinations in Multiple Myeloma Monday, December 9, 2024 4:30 PM PST Venclexta ® (venetoclax)* CRISTALLO: Results from a Phase III Trial of Venetoclax–Obinutuzumab versus Fludarabine, Cyclophosphamide and Rituximab or Bendamustine–Rituximab in Patients with Untreated Chronic Lymphocytic Leukemia Without Del(17p) or TP53 Mutations #3237 poster presentation Session: 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster II Sunday, December 8, 2024 6:00 - 8:00 PM PST P-CD19CD20-ALLO1 (in collaboration with Poseida Therapeutics) P-CD19CD20-ALLO1: Potent Fully Allogeneic CAR-T Therapy Targeting CD19 and CD20 with Superior Efficacy Over Single-Target Products #4805 poster presentation Session: 702. CAR-T Cell Therapies: Basic and Translational: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST P-BCMA-ALLO1 (in collaboration with Poseida Therapeutics) A Phase 1 Study of P-BCMA-ALLO1, a Non-viral, Allogeneic BCMA Directed CAR-T in Relapsed/Refractory Multiple Myeloma (RRMM): Results from Optimized Lymphodepletion Cohort #4828 poster presentation Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST Hemlibra ® (emicizumab) Bleed Patterns in Infants, From Birth to 12 Months of Age, with Hemophilia A Treated with Emicizumab: Exploratory Analysis of the HAVEN 7 Study #1214 poster presentation Session: 322. Hemophilia A and B: Clinical and Epidemiological: Poster I Saturday, December 7, 2024 5:30 - 7:30 PM PST Real-World Experience With Emicizumab for Hemophilia A From the Physician Perspective Based on Survey Data #5078 poster presentation Session: 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST PiaSky ® (crovalimab) Phase III Randomized COMMODORE 2 Trial: 2-Year Efficacy and Safety of Crovalimab in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Naive to Complement Inhibition #2687 poster presentation Session: 508. Bone Marrow Failure: Acquired: Poster II Sunday, December 8, 2024 6:00 - 8:00 PM PST Phase III COMMODORE 1 Trial: 2-Year Efficacy and Safety of Crovalimab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Switched from Ravulizumab #4078 poster presentation Session: 508. Bone Marrow Failure: Acquired: Poster III Monday, December 9, 2024 6:00 - 8:00 PM PST *Venclexta is being developed by AbbVie and Genentech, a member of the Roche Group. It is jointly commercialized by the companies in the United States and commercialized by AbbVie outside of the United States. About Polivy ® (polatuzumab vedotin-piiq) Polivy is a first-in-class anti-CD79b antibody-drug conjugate (ADC). The CD79b protein is expressed specifically in the majority of B cells, an immune cell impacted in some types of non-Hodgkin’s lymphoma (NHL), making it a promising target for the development of new therapies. Polivy binds to cancer cells such as CD79b and destroys these B cells through the delivery of an anti-cancer agent, which is thought to minimize the effects on normal cells. Polivy is being developed by Genentech using Seagen ADC technology and is currently being investigated for the treatment of several types of NHL. Polivy U.S. Indication Polivy is a prescription medicine used with other medicines (a rituximab product, cyclophosphamide, doxorubicin, and prednisone) as a first treatment for adults who have moderate to high risk diffuse large B-cell lymphoma (DLBCL), not otherwise specified (NOS) or high-grade B-cell lymphoma (HGBL). Polivy is a prescription medicine used with other medicines, bendamustine and a rituximab product, to treat DLBCL in adults who have progressed after at least 2 prior therapies. Important Safety Information Possible serious side effects Everyone reacts differently to Polivy therapy, so it’s important to know what the side effects are. Some people who have been treated with Polivy have experienced serious to fatal side effects. Your doctor may stop or adjust your treatment if any serious side effects occur. Be sure to contact your healthcare team if there are any signs of these side effects. Nerve problems in your arms and legs: This may happen as early as after your first dose and may worsen with every dose. Your doctor will monitor for signs and symptoms, such as changes in your sense of touch, numbness or tingling in your hands or feet, nerve pain, burning sensation, any muscle weakness, or changes to your walking pattern Infusion-related reactions: You may experience fever, chills, rash, breathing problems, low blood pressure, or hives within 24 hours of your infusion Low blood cell counts: Treatment with Polivy can cause severe low blood cell counts. Your doctor will monitor your blood counts throughout treatment with Polivy Infections: If you have a fever of 100.4°F (38°C) or higher, chills, cough, or pain during urination, contact your healthcare team. Your doctor may also give you medication before giving you Polivy, which may prevent some infections Rare and serious brain infections: Your doctor will monitor closely for signs and symptoms of these types of infections. Contact your doctor if you experience confusion, dizziness or loss of balance, trouble talking or walking, or vision changes Tumor lysis syndrome: Caused by the fast breakdown of cancer cells. Signs include nausea, vomiting, diarrhea, and lack of energy Potential harm to liver: Some signs include tiredness, weight loss, pain in the abdomen, dark urine, and yellowing of your skin or the white part of your eyes. You may be at higher risk if you already had liver problems or you are taking other medication Side effects seen most often The most common side effects during treatment were Nerve problems in arms and legs Nausea Tiredness or lack of energy Diarrhea Constipation Hair loss Redness and sores of the lining of the mouth, lips, throat, and digestive tract Polivy may lower your red or white blood cell counts and increase uric acid levels. Polivy may not be for everyone. Talk to your doctor if you are Pregnant or think you are pregnant: Data have shown that Polivy may harm your unborn baby Planning to become pregnant: Women should avoid getting pregnant while taking Polivy. Women should use effective contraception during treatment and for 3 months after their last Polivy treatment. Men taking Polivy should use effective contraception during treatment and for 5 months after their last Polivy treatment Breastfeeding: Women should not breastfeed while taking Polivy and for 2 months after the last dose These may not be all the side effects. Talk to your healthcare provider for more information about the benefits and risks of Polivy treatment. You may report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch . You may also report side effects to Genentech at (888) 835-2555. Please see the full Prescribing Information and visit https://www.Polivy.com for additional Important Safety Information. About Lunsumio ® (mosunetuzumab-axgb) Lunsumio is a first-in-class CD20xCD3 T-cell engaging bispecific antibody designed to target CD20 on the surface of B cells and CD3 on the surface of T cells. This dual targeting activates and redirects a patient’s existing T cells to engage and eliminate target B cells by releasing cytotoxic proteins into the B cells. A robust clinical development program for Lunsumio is ongoing, investigating the molecule as a monotherapy and in combination with other medicines, for the treatment of people with B-cell non-Hodgkin’s lymphomas, including follicular lymphoma and diffuse large B-cell lymphoma, and other blood cancers. Lunsumio U.S. Indication Lunsumio (mosunetuzumab-axgb) is a prescription medicine used to treat adults with follicular lymphoma whose cancer has come back or did not respond to previous treatment, and who have already received two or more treatments for their cancer. It is not known if Lunsumio is safe and effective in children. The conditional approval of Lunsumio is based on response rate. There are ongoing studies to establish how well the drug works. What is the most important information I should know about Lunsumio? Lunsumio may cause Cytokine Release Syndrome (CRS), a serious side effect that is common during treatment with Lunsumio and can also be severe or life-threatening. Get medical help right away if you develop any signs or symptoms of CRS at any time, including: fever of 100.4°F (38°C) or higher chills low blood pressure fast or irregular heartbeat tiredness or weakness difficulty breathing headache confusion feeling anxious dizziness or light-headedness nausea vomiting Due to the risk of CRS, you will receive Lunsumio on a “step-up dosing schedule.” The step-up dosing schedule is when you receive smaller “step-up” doses of Lunsumio on Day 1 and Day 8 of your first cycle of treatment You will receive a higher dose of Lunsumio on Day 15 of your first cycle of treatment If your dose of Lunsumio is delayed for any reason, you may need to repeat the step-up dosing schedule Before each dose in Cycle 1 and Cycle 2, you will receive medicines to help reduce your risk of CRS Your healthcare provider will check you for CRS during treatment with Lunsumio and may treat you in a hospital if you develop signs and symptoms of CRS. Your healthcare provider may temporarily stop or completely stop your treatment with Lunsumio, if you have severe side effects. What are the possible side effects of Lunsumio? Lunsumio may cause serious side effects, including: Neurologic problems. Your healthcare provider will check you for neurologic problems during treatment with Lunsumio. Your healthcare provider may also refer you to a healthcare provider who specializes in neurologic problems. Tell your healthcare provider right away if you develop any signs or symptoms of neurologic problems during or after treatment with Lunsumio, including: headache numbness and tingling of the arms, legs, hands, or feet dizziness confusion and disorientation difficulty paying attention or understanding things forgetting things or forgetting who or where you are trouble speaking, reading, or writing sleepiness or trouble sleeping tremors loss of consciousness seizures muscle problems or muscle weakness loss of balance or trouble walking Serious infections. Lunsumio can cause serious infections that may lead to death. Your healthcare provider will check you for signs and symptoms of infection before and during treatment. Tell your healthcare provider right away if you develop any signs or symptoms of infection during treatment with Lunsumio, including: fever of 100.4° F (38° C) or higher chest pain tiredness shortness of breath painful rash sore throat pain during urination feeling weak or generally unwell Low blood cell counts. Low blood cell counts are common during treatment with Lunsumio and can also be severe. Your healthcare provider will check your blood cell counts during treatment with Lunsumio. Lunsumio may cause the following low blood cell counts: low white blood cell counts (neutropenia). Low white blood cells can increase your risk for infection low red blood cell counts (anemia). Low red blood cells can cause tiredness and shortness of breath low platelet counts (thrombocytopenia). Low platelet counts can cause bruising or bleeding problems Growth in your tumor or worsening of tumor related problems (Tumor flare). Lunsumio may cause serious or severe worsening of your tumor. Tell your healthcare provider if you develop any of these signs or symptoms of tumor flare during your treatment with Lunsumio: tender or swollen lymph nodes, chest pain, cough, trouble breathing, and pain or swelling at the site of the tumor Your healthcare provider may temporarily stop or permanently stop treatment with Lunsumio if you develop severe side effects. The most common side effects of Lunsumio include: tiredness, rash, fever, and headache. The most common severe abnormal lab test results with Lunsumio include: decreased phosphate, increased glucose, and increased uric acid levels. Before receiving Lunsumio, tell your healthcare provider about all of your medical conditions, including if you: have ever had an infusion reaction after receiving Lunsumio have an infection, or have had an infection in the past which lasted a long time or keeps coming back have or have had Epstein-Barr Virus are pregnant or plan to become pregnant. Lunsumio may harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Lunsumio Females who are able to become pregnant: your healthcare provider should do a pregnancy test before you start treatment with Lunsumio you should use an effective method of birth control during your treatment and for 3 months after the last dose of Lunsumio are breastfeeding or plan to breastfeed. It is not known if Lunsumio passes into your breast milk. Do not breastfeed during treatment and for 3 months after the last dose of Lunsumio Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. What should I avoid while receiving Lunsumio? Do not drive, operate heavy machinery, or do other dangerous activities if you develop dizziness, confusion, tremors, sleepiness, or any other symptoms that impair consciousness until your signs and symptoms go away. These may be signs and symptoms of CRS or neurologic problems. These are not all the possible side effects of Lunsumio. Talk to your healthcare provider for more information about the benefits and risks of Lunsumio. You may report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch . You may also report side effects to Genentech at (888) 835-2555. Please see Important Safety Information, including Serious Side Effects, as well as the Lunsumio full Prescribing Information and Medication Guide or visit https://www.Lunsumio.com . About Columvi ® (glofitamab-gxbm) Columvi is a CD20xCD3 T-cell engaging bispecific antibody designed to target CD3 on the surface of T cells and CD20 on the surface of B cells. Columvi was designed with a novel 2:1 structural format. This T-cell engaging bispecific antibody is engineered to have one region that binds to CD3, a protein on T cells, a type of immune cell, and two regions that bind to CD20, a protein on B cells, which can be healthy or malignant. This dual-targeting brings the T cell in close proximity to the B cell, activating the release of cancer cell-killing proteins from the T cell. A clinical development program for Columvi is ongoing, investigating the molecule as a monotherapy and in combination with other medicines for the treatment of people with B-cell non-Hodgkin’s lymphomas, including diffuse large B-cell lymphoma and other blood cancers. Columvi U.S. Indication Columvi (glofitamab-gxbm) is a prescription medicine to treat adults with certain types of diffuse large B-cell lymphoma (DLBCL) or large B-cell lymphoma (LBCL) that has come back (relapsed) or that did not respond to previous treatment (refractory), and who have received 2 or more prior treatments for their cancer. It is not known if Columvi is safe and effective in children. The conditional approval of Columvi is based on response rate and durability of response. There are ongoing studies to establish how well the drug works. What is the most important information I should know about Columvi? Columvi can cause Cytokine Release Syndrome (CRS), a serious side effect that is common during treatment with Columvi, and can also be serious and lead to death. Call your healthcare provider or get emergency medical help right away if you develop any signs or symptoms of CRS, including: fever of 100.4°F (38°C) or higher chills or shaking fast or irregular heartbeat dizziness or light-headedness trouble breathing shortness of breath Due to the risk of CRS, you will receive Columvi on a “step-up dosing schedule”. A single dose of a medicine called obinutuzumab will be given to you on the first day of your first treatment cycle (Day 1 of Cycle 1). You will start the Columvi step-up dosing schedule a week after the obinutuzumab dose. The step-up dosing schedule is when you receive smaller “step-up” doses of Columvi on Day 8 and Day 15 of Cycle 1. This is to help reduce your risk of CRS. You should be hospitalized during your infusion and for 24 hours after receiving the first step-up dose on Day 8. You should be hospitalized during your infusion and for 24 hours after receiving the second step-up dose on Day 15 if you experienced CRS during the first step-up dose. You will receive your first full dose of Columvi a week after the second step-up dose (this will be Day 1 of Cycle 2). If your dose of Columvi is delayed for any reason, you may need to repeat the “step-up dosing schedule”. If you had more than mild CRS with your previous dose of Columvi, you should be hospitalized during and for 24 hours after receiving your next dose of Columvi. Before each dose of Columvi, you will receive medicines to help reduce your risk of CRS and infusion-related reactions. Your healthcare provider will monitor you for CRS during treatment with Columvi and may treat you in a hospital if you develop signs and symptoms of CRS. Your healthcare provider may temporarily stop or completely stop your treatment with Columvi if you have severe side effects. Carry the Columvi Patient Wallet Card with you at all times and show it to all of your healthcare providers. The Columvi Patient Wallet Card lists the signs and symptoms of CRS you should get emergency medical help for right away. What are the possible side effects of Columvi? Columvi may cause serious side effects, including: Cytokine Release Syndrome. Neurologic problems. Columvi can cause serious neurologic problems that may lead to death. Your healthcare provider will monitor you for neurologic problems during treatment with Columvi. Your healthcare provider may also refer you to a healthcare provider who specializes in neurologic problems. Tell your healthcare provider right away if you develop any signs or symptoms of neurologic problems, including: headache confusion and disorientation difficulty paying attention or understanding things trouble speaking sleepiness memory problems numbness, tingling, or weakness of the hands or feet dizziness shaking (tremors) Serious Infections. Columvi can cause serious infections that may lead to death. Your healthcare provider will monitor you for signs and symptoms of infection and treat you as needed. Tell your healthcare provider right away if you develop any signs of an infection, including: fever, chills, weakness, cough, shortness of breath, or sore throat. Growth in your tumor or worsening of tumor related problems (tumor flare). Tell your healthcare provider if you get any of these signs or symptoms of tumor flare: tender or swollen lymph nodes pain or swelling at the site of the tumor chest pain cough trouble breathing The most common side effects of Columvi include: CRS, muscle and bone pain, rash, and tiredness. The most common severe abnormal lab test results with Columvi include: decreased white blood cells, decreased phosphate (an electrolyte), increased uric acid levels, and decreased fibrinogen (a protein that helps with blood clotting). Your healthcare provider may temporarily stop or completely stop treatment with Columvi if you develop certain side effects. Before receiving Columvi, tell your healthcare provider about all of your medical conditions, including if you: have an infection have kidney problems are pregnant or plan to become pregnant. Columvi may harm your unborn baby Females who are able to become pregnant: Your healthcare provider should do a pregnancy test before you start treatment with Columvi. You should use effective birth control (contraception) during treatment and for 1 month after your last dose of Columvi. Talk to your healthcare provider about what birth control method is right for you during this time. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Columvi. are breastfeeding or plan to breastfeed. Columvi may pass into your breast milk. Do not breastfeed during treatment and for 1 month after your last dose of Columvi. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. What should I avoid while receiving Columvi? Do not drive, operate heavy machinery, or do other dangerous activities if you develop dizziness, confusion, shaking (tremors), sleepiness, or any other symptoms that impair consciousness until your signs and symptoms go away. These may be signs and symptoms of neurologic problems. These are not all the possible side effects of Columvi. Talk to your health care provider for more information about the benefits and risks of Columvi. You may report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch . You may also report side effects to Genentech at (888) 835-2555. Please see Important Safety Information, including Serious Side Effects , as well as the Columvi full Prescribing Information and Medication Guide or visit https://www.Columvi.com . About Venclexta ® (venetoclax) Venclexta is a first-in-class targeted medicine designed to selectively bind and inhibit the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers and other tumors, BCL-2 builds up and prevents cancer cells from dying or self-destructing, a process called apoptosis. Venclexta blocks the BCL-2 protein and works to help restore the process of apoptosis. Venclexta is being developed by AbbVie and Genentech, a member of the Roche Group. It is jointly commercialized by the companies in the United States and commercialized by AbbVie outside of the United States. Together, the companies are committed to research with Venclexta, which is currently being studied in clinical trials across several types of blood cancers. Venclexta U.S. Indications Venclexta is a prescription medicine used: to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). in combination with azacitidine, or decitabine, or low-dose cytarabine to treat adults with newly-diagnosed acute myeloid leukemia (AML) who: ‒ are 75 years of age or older, or ‒ have other medical conditions that prevent the use of standard chemotherapy. It is not known if Venclexta is safe and effective in children. Important Safety Information What is the most important information patients should know about Venclexta? Venclexta can cause serious side effects, including: Tumor lysis syndrome (TLS). TLS is caused by the fast breakdown of cancer cells. TLS can cause kidney failure, the need for dialysis treatment, and may lead to death. The patient’s doctor will do tests to check their risk of getting TLS before they start taking Venclexta. The patient will receive other medicines before starting and during treatment with Venclexta to help reduce the risk of TLS. The patient may also need to receive intravenous (IV) fluids into their vein. The patient’s doctor will do blood tests to check for TLS when the patient first starts treatment and during treatment with Venclexta. It is important for patients to keep appointments for blood tests. Patients should tell their doctor right away if they have any symptoms of TLS during treatment with Venclexta, including fever, chills, nausea, vomiting, confusion, shortness of breath, seizures, irregular heartbeat, dark or cloudy urine, unusual tiredness, or muscle or joint pain. Patients should drink plenty of water during treatment with Venclexta to help reduce the risk of getting TLS. Patients should drink 6 to 8 glasses (about 56 ounces total) of water each day, starting 2 days before the first dose on the day of the first dose of Venclexta, and each time a dose is increased. The patient’s doctor may delay, decrease the dose, or stop treatment with Venclexta if the patient has side effects. When restarting Venclexta after stopping for 1 week or longer, the patient’s doctor may again check for the risk of TLS and change the patient’s dose. What patients should not take Venclexta? Certain medicines must not be taken when the patient first starts taking Venclexta and while the dose is being slowly increased because of the risk of increased TLS. Patients should tell their doctor about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Venclexta and other medicines may affect each other causing serious side effects. Patients must not start new medicines during treatment with Venclexta without first talking with their doctor. Before taking Venclexta, patients must tell their doctor about all of their medical conditions, including if they: Have kidney or liver problems. Have problems with body salts or electrolytes, such as potassium, phosphorus, or calcium. Have a history of high uric acid levels in the blood or gout. Are scheduled to receive a vaccine. Patients should not receive a “live vaccine” before, during, or after treatment with Venclexta, until the patient’s doctor tells them it is okay. If the patient is not sure about the type of immunization or vaccine, the patient should ask their doctor. These vaccines may not be safe or may not work as well during treatment with Venclexta. Are pregnant or plan to become pregnant. Venclexta may harm an unborn baby. If the patient is able to become pregnant, the patient’s doctor should do a pregnancy test before the patient starts treatment with Venclexta, and the patient should use effective birth control during treatment and for at least 30 days after the last dose of Venclexta. If the patient becomes pregnant or thinks they are pregnant, the patient should tell their doctor right away. Are breastfeeding or plan to breastfeed. It is not known if Venclexta passes into the patient’s breast milk. Patients are instructed to not breastfeed during treatment with Venclexta and for 1 week after the last dose. What to avoid while taking Venclexta: Patients should not drink grapefruit juice or eat grapefruit, Seville oranges (often used in marmalades), or starfruit while they are taking Venclexta. These products may increase the amount of Venclexta in the patient’s blood. What are the possible side effects of Venclexta? Venclexta can cause serious side effects, including: Low white blood cell counts (neutropenia). Low white blood cell counts are common with Venclexta, but can also be severe. The patient’s doctor will do blood tests to check their blood counts during treatment with Venclexta and may pause dosing. Infections. Death and serious infections such as pneumonia and blood infection (sepsis) have happened during treatment with Venclexta. The patient’s doctor will closely monitor and treat the patient right away if they have a fever or any signs of infection during treatment with Venclexta. Patients should tell their doctor right away if they have a fever or any signs of an infection during treatment with Venclexta. The most common side effects of Venclexta when used in combination with obinutuzumab or rituximab or alone in people with CLL or SLL include low white blood cell count; low platelet count; low red blood cell count; diarrhea; nausea; upper respiratory tract infection; cough; muscle and joint pain; tiredness; and swelling of arms, legs, hands, and feet. The most common side effects of Venclexta in combination with azacitidine or decitabine or low-dose cytarabine in people with AML include nausea; diarrhea; low platelet count; constipation; low white blood cell count; fever with low white blood cell count; tiredness; vomiting; swelling of arms, legs, hands, or feet; fever; infection in lungs; shortness of breath; bleeding; low red blood cell count; rash; stomach (abdominal) pain; infection in your blood; muscle and joint pain; dizziness; cough; sore throat; and low blood pressure. Venclexta may cause fertility problems in males. This may affect the ability to father a child. Patients should talk to their doctor if they have concerns about fertility. These are not all the possible side effects of Venclexta. Patients should call their doctor for medical advice about side effects. Report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch . Report side effects to Genentech at 1-888-835-2555. Please see the Venclexta full Prescribing Information , including the Medication Guide , for additional Important Safety Information or visit https://www.venclexta.com/ . About Hemlibra Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins required to activate the natural coagulation cascade and restore the blood clotting process for hemophilia A patients. Hemlibra is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once weekly, every two weeks or every four weeks. Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed globally by Chugai, Roche and Genentech . Hemlibra U.S. Indication Hemlibra is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A with or without factor VIII inhibitors. Important Safety Information What is the most important information to know about Hemlibra? Hemlibra increases the potential for blood to clot. People who use activated prothrombin complex concentrate (aPCC; Feiba ® ) to treat breakthrough bleeds while taking Hemlibra may be at risk of serious side effects related to blood clots. These serious side effects include: Thrombotic microangiopathy (TMA), a condition involving blood clots and injury to small blood vessels that may cause harm to one’s kidneys, brain, and other organs Blood clots (thrombotic events), which may form in blood vessels in the arm, leg, lung, or head Patients should talk to their doctor about the signs and symptoms of these serious side effects, which can include Confusion Stomach, chest, or back pain Weakness Nausea or vomiting Swelling, pain, or redness Feeling sick or faint Decreased urination Swelling of arms and legs Yellowing of skin and eyes Eye pain, swelling, or trouble seeing Fast heart rate Numbness in your face Headache Shortness of breath Coughing up blood If patients experience any of these symptoms during or after treatment with Hemlibra, they should get medical help right away. Patients should carefully follow their healthcare provider’s instructions regarding when to use an on demand bypassing agent or factor VIII, and the dose and schedule to use for breakthrough bleed treatment. If aPCC (Feiba ® ) is needed, patients should talk to their healthcare provider in case they feel they need more than 100 U/kg of aPCC (Feiba ® ) total. Patients’ bodies may make antibodies against Hemlibra, which may stop Hemlibra from working properly. Patients should contact their healthcare provider immediately if they notice that Hemlibra has stopped working for them (e.g., increase in bleeds). The most common side effects of Hemlibra include: injection site reactions (redness, tenderness, warmth, or itching at the site of injection), headache, and joint pain. These are not all of the possible side effects of Hemlibra. Patients can speak with their healthcare provider for more information. What else should patients know about Hemlibra? Patients should see the detailed “Instructions for Use” that comes with Hemlibra for information on how to prepare and inject a dose of Hemlibra, and how to properly throw away (dispose of) used needles and syringes. Patients should stop taking their prophylactic bypassing therapy the day before they start Hemlibra Patients may continue taking their prophylactic factor VIII for the first week of Hemlibra Hemlibra may interfere with laboratory tests that measure how well blood is clotting and create an inaccurate result. Patients should speak with their healthcare provider about how this may affect their care. Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. Patients should only use Hemlibra for the condition it was prescribed. Patients should not give Hemlibra to other people, even if they have the same symptoms that they have. It may harm them. Patients should tell their healthcare provider about all the medicines they take , including prescription medicines, over-the-counter medicines, vitamins, or herbal supplements. Patients should keep a list of them to show their healthcare provider and pharmacist. Before using Hemlibra, patients should tell their healthcare provider about all of their medical conditions , including if they are pregnant, plan to become pregnant, are breastfeeding, or plan to breastfeed. Since Hemlibra was tested in males, there is no information on whether Hemlibra may impact an unborn baby or breast milk. Females who are able to become pregnant should use birth control during treatment. Side effects may be reported to the FDA at (800) FDA-1088 or www.fda.gov/medwatch . Side effects may also be reported to Genentech at (888) 835-2555. Please see Important Safety Information, including Serious Side Effects, as well as the Hemlibra full Prescribing Information and Medication Guide or visit https://www.hemlibra.com/ . Indications & Important Safety Information What is PIASKY? PIASKY is a prescription medicine used to treat a disease called paroxysmal nocturnal hemoglobinuria (PNH) in adults and children 13 years of age or older who weigh at least 88 pounds (40 kg). It is not known if PIASKY is safe and effective in children under 13 years of age and in people who weigh less than 88 pounds (40kg). What is the most important information I should know about PIASKY? PIASKY is a medicine that can affect your immune system. PIASKY may lower the ability of your immune system to fight infections PIASKY increases your chance of getting serious infections caused by Neisseria meningitidis . Meningococcal infections may quickly become life-threatening or cause death if not recognized and treated early. You must complete or update your meningococcal vaccines at least 2 weeks before your first dose of PIASKY. If your healthcare provider decides that immediate treatment with PIASKY is needed and your meningococcal vaccination is not up to date, you should receive meningococcal vaccination as soon as possible, and receive antibiotics for as long as your healthcare provider tells you. If you have been given a meningococcal vaccine in the past, you might need additional vaccines before starting PIASKY. Your healthcare provider will decide if you need additional meningococcal vaccine. Meningococcal vaccines do not prevent all meningococcal infections. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a serious meningococcal infection: fever fever and a rash fever with a high heart rate fever with a headache headache with nausea or vomiting headache with a stiff neck or stiff back confusion muscle aches, with flu-like symptoms eyes sensitive to light Your healthcare provider will give you a Patient Safety Card about the risk of serious meningococcal infection. Carry it with you at all times during treatment and for 11 months after your last dose of PIASKY. Your risk of meningococcal infection may continue for several months after your last dose of PIASKY. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly. PIASKY is only available through a program called the PIASKY Risk Evaluation and Mitigation Strategy (PIASKY REMS). Before you can receive PIASKY, your healthcare provider must: enroll in the PIASKY REMS program. counsel you about the risk of serious meningococcal infection. give you information about the signs and symptoms of serious meningococcal infection. make sure that you are vaccinated with a meningococcal vaccine and that you receive antibiotics if you need to start PIASKY right away if you are not up to date on your vaccines. give you a Patient Safety Card about your risk of meningococcal infection. Immune system reactions called Type III hypersensitivity reactions are common during treatment with PIASKY and can be serious. If you are currently being treated with or have been treated with another C5 inhibitor medicine and you switch to PIASKY , PIASKY may cause Type III hypersensitivity reactions. People may also develop Type III hypersensitivity reactions when they switch from PIASKY to another C5 inhibitor medicine. If you have been treated with another C5 inhibitor medicine and you switch to PIASKY, or if you have been treated with PIASKY and you switch to another C5 inhibitor medicine, your healthcare provider should monitor you for 30 days after you switch medicines. Call your healthcare provider or go to the nearest emergency room right away if you have any signs or symptoms of Type III hypersensitivity reaction including: joint pain muscle or bone pain rash or skin problems itching headache kidney problems numbness and tingling or a feeling of pins and needles especially of the hands and feet fever weakness, tiredness, or lack of energy stomach trouble or pain PIASKY may also increase the risk of other types of serious infections , including infections caused by Neisseria spp. , Streptococcus pneumoniae , Haemophilus influenzae , and Neisseria gonorrhoeae . If you receive treatment with PIASKY, you should receive vaccines against Streptococcus pneumoniae . If your child receives treatment with PIASKY, your child should receive vaccines against Streptococcus pneumoniae and may receive vaccines against Haemophilus influenzae , depending on their age. Call your healthcare provider right away if you have any new signs or symptoms of infection such as: fever of 100.4°F (38°C) or higher cough chest pain tiredness feeling short of breath painful rash sore throat burning pain when passing urine feeling weak or generally unwell Who should not receive PIASKY? Do not receive PIASKY if you: Have a serious meningococcal infection caused by Neisseria meningitidis when you are starting PIASKY treatment. Are allergic to crovalimab or any of the ingredients in PIASKY. Before receiving PIASKY tell your healthcare provider about all of your medical conditions, including if you: have an infection or fever. are pregnant or plan to become pregnant. It is not known if PIASKY may harm your unborn baby. are breastfeeding or plan to breastfeed. It is not known if PIASKY passes into your breast milk. Tell your healthcare provider about all the medicines you take , including prescription and over-the-counter medicines, vitamins, and herbal supplements. PIASKY and other medicines can affect each other, causing side effects. Especially tell your healthcare provider if you are currently being treated with or have ever been treated with any other complementary C5 inhibitor (C5 inhibitor) medicine. PIASKY is a C5 inhibitor medicine. Know the medicines you take and the vaccines you receive. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine. How should I receive PIASKY? Your healthcare provider will give you your PIASKY treatment. Your first dose will be given through a vein by intravenous (IV) infusion on Day 1 by your healthcare provider. This is the first loading dose. Another loading dose will be given as an injection under the skin (subcutaneous) on Days 2, 8, 15, and 22. Your maintenance doses will begin on Day 29 and then will be given every 4 weeks as a subcutaneous injection. Your healthcare provider will prescribe the dose based on your weight. If your weight changes, tell your healthcare provider. Talk to your healthcare provider if you miss receiving your dose of PIASKY. If you are changing treatment from another C5 inhibitor such as eculizumab or ravulizumab to PIASKY , you should receive your first loading dose of PIASKY no sooner than the time you would have received your next scheduled dose of eculizumab or ravulizumab. If you stop taking PIASKY and do not switch to another treatment for your PNH, your healthcare provider will need to monitor you closely for at least 20 weeks after stopping PIASKY. Stopping treatment with PIASKY may cause a breakdown of red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: a lower number of red blood cells (anemia) blood in your urine or dark urine feeling short of breath feeling tired or low energy (fatigue) stomach pain blood clotting (thrombosis) difficulty swallowing difficulty getting or keeping an erection (erectile dysfunction) kidneys not working properly What are the possible side effects of PIASKY? PIASKY can cause serious side effects including: Infusion- and injection-related reactions. Infusion- or injection-related reactions may happen during or after your PIASKY administration. Symptoms may include headache, pain at infusion or injection site or in other parts of your body, swelling, bruising or bleeding, red skin, itching and rash. PIASKY can also cause serious allergic reactions. Tell your healthcare provider right away or go to the nearest emergency room if you get any of the following symptoms or symptoms of a serious allergic reaction: shortness of breath or trouble breathing pain or tightness in your chest wheezing feeling dizzy or lightheaded swelling of the throat, lips, tongue, or face skin itching, hives, or rash fever or chills The most common side effects of PIASKY are: infusion-related reactions respiratory tract infections including infections of the lungs, cold symptoms, and pain or swelling of the nose or throat viral infections Type III hypersensitivity reactions Tell your healthcare provider about any side effect that bothers you or that does not go away. These are not all the possible side effects of PIASKY. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . You may also report side effects to Genentech at (888) 835-2555. Please see the full Prescribing Information and Medication Guide for additional Important Safety Information, including Serious Side Effects , or visit https://www.piasky.com/ . About Genentech in Hematology For more than 20 years, Genentech has been developing medicines with the goal to redefine treatment in hematology. Today, we’re investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. For more information visit http://www.gene.com/hematology . About Genentech Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com .

Phase 2Clinical ResultPhase 3Phase 1ASH

09 Dec 2023

·www.businesswire.com

New Data Reinforce the Benefit of Early Preventative Treatment With Genentech’s Hemlibra (emicizumab-kxwh) for Babies With Severe hemophilia A

SOUTH SAN FRANCISCO, Calif.--( BUSINESS WIRE )--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the primary analysis of the Phase III HAVEN 7 study reinforced the efficacy and safety of Hemlibra ® (emicizumab-kxwh) in previously untreated or minimally treated infants with severe hemophilia A without factor VIII inhibitors. Results showed that Hemlibra achieved meaningful bleed control in babies up to 12 months of age and was well tolerated. The new data were presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition taking place December 9-12, 2023 in San Diego, California and included in the press program. “Hemophilia A can have a devastating impact on any patient, but this is especially true for infants, where the emotional and physical stress due to frequent hospital visits, treatment administration and other worries can be distressing for babies and their parents and caregivers,” said Steven Pipe, M.D., professor of pediatrics and pathology at the University of Michigan. “These results reinforce the benefit of starting prophylaxis as soon as possible after birth, as well as for the use of subcutaneous treatments, which are especially valuable in young babies where access to veins can be very difficult.” The burden of severe hemophilia A in babies and on their parents and caregivers is significant. The World Federation of Hemophilia treatment guidelines consider the standard of care in hemophilia to be regular prophylaxis initiated at a young age, as studies have shown this improves long-term outcomes, while reducing the risk of intracranial hemorrhage. However, for many babies with hemophilia A, prophylaxis is not started until after the first year of life because of the high treatment burden. Hemlibra, which is already approved and being used to treat babies with hemophilia A, provides a flexible treatment option that can be administered subcutaneously from birth at different dosing frequencies. “The results of HAVEN 7 provide additional confidence in the efficacy and safety profile of Hemlibra for babies with severe hemophilia A and add to its extensive clinical and real-world evidence across all ages,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “Conducted in collaboration with the hemophilia A community, this trial reflects our ongoing commitment to listen and respond to the needs of those impacted by this condition in hopes of advancing treatment standards even further.” The HAVEN 7 study is a Phase III, descriptive, single-arm study set up in collaboration with the hemophilia A community to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of subcutaneous Hemlibra in infants with severe hemophilia A without factor VIII inhibitors. These results, which included data from 55 participants, showed that at 101.9 weeks median follow-up, 54.5% of participants (n=30) did not have any bleeds that required treatment, while 16.4% (n=9) did not have any treated or untreated bleeds at all. There were no spontaneous bleeds requiring treatment in any participant, and all treated bleeds were as a result of trauma. A total of 207 bleeds occurred in 46 participants (83.6%); 87.9% of these were as a result of trauma. The model-based annualized bleeding rate (95% CI) was 0.4 (0.30–0.63) for treated bleeds. No new safety signals were observed and there were no treatment-related serious adverse events, intracranial hemorrhages or deaths reported. Just 3.6% of participants (n=2) tested positive for factor VIII inhibitors, which may be a consequence of reduced factor VIII usage in participants treated with Hemlibra, and no participant tested positive for anti-drug antibodies. Results were consistent with positive results from the interim analysis and from previous Phase III HAVEN studies. The results of additional research on biomarkers in the HAVEN 7 study were also presented at ASH and were supportive of the study’s primary efficacy analysis. This additional research showed that the pharmacodynamic profiles of Hemlibra in babies were consistent with those previously observed in older children and adults with hemophilia A. The data showed that Hemlibra exhibits the expected pharmacodynamic response despite the reduced presence of the clotting factors that Hemlibra binds to in this age group. The Phase III HAVEN 7 study results complement data from the broader, pivotal HAVEN clinical program, providing insights into the evolution of hemophilia A in babies, and the impact of initiating preventative treatment from birth. The primary analysis is being followed by a seven-year extension period. Hemlibra continues to redefine standards of care in hemophilia A as a flexible treatment option approved across all ages and stages of life regardless of inhibitor status and at different dosing frequencies. It is approved for the routine prophylaxis of people with hemophilia A in more than 115 countries worldwide. It has been studied in one of the largest clinical trial programs in people with hemophilia A with and without factor VIII inhibitors, including eight Phase III studies. About Hemlibra Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins required to activate the natural coagulation cascade and restore the blood clotting process for hemophilia A patients. Hemlibra is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once weekly, every two weeks or every four weeks. Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed globally by Chugai, Roche and Genentech. Hemlibra U.S. Indication Hemlibra is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A with or without factor VIII inhibitors. Important Safety Information What is the most important information to know about Hemlibra? Hemlibra increases the potential for blood to clot. People who use activated prothrombin complex concentrate (aPCC; Feiba ® ) to treat breakthrough bleeds while taking Hemlibra may be at risk of serious side effects related to blood clots. These serious side effects include: Thrombotic microangiopathy (TMA), a condition involving blood clots and injury to small blood vessels that may cause harm to one’s kidneys, brain, and other organs Blood clots (thrombotic events), which may form in blood vessels in the arm, leg, lung, or head Patients should talk to their doctor about the signs and symptoms of these serious side effects, which can include Confusion Stomach, chest, or back pain Weakness Nausea or vomiting Swelling, pain, or redness Feeling sick or faint Decreased urination Swelling of arms and legs Yellowing of skin and eyes Eye pain, swelling, or trouble seeing Fast heart rate Numbness in your face Headache Shortness of breath Coughing up blood If patients experience any of these symptoms during or after treatment with Hemlibra, they should get medical help right away. Patients should carefully follow their healthcare provider’s instructions regarding when to use an on demand bypassing agent or factor VIII, and the dose and schedule to use for breakthrough bleed treatment. If aPCC (Feiba ® ) is needed, patients should talk to their healthcare provider in case they feel they need more than 100 U/kg of aPCC (Feiba ® ) total. Patients’ bodies may make antibodies against Hemlibra, which may stop Hemlibra from working properly. Patients should contact their healthcare provider immediately if they notice that Hemlibra has stopped working for them (e.g., increase in bleeds). The most common side effects of Hemlibra include : injection site reactions (redness, tenderness, warmth, or itching at the site of injection), headache, and joint pain. These are not all of the possible side effects of Hemlibra. Patients can speak with their healthcare provider for more information. What else should patients know about Hemlibra? Patients should see the detailed “Instructions for Use” that comes with Hemlibra for information on how to prepare and inject a dose of Hemlibra, and how to properly throw away (dispose of) used needles and syringes. Patients should stop taking their prophylactic bypassing therapy the day before they start Hemlibra Patients may continue taking their prophylactic factor VIII for the first week of Hemlibra Hemlibra may interfere with laboratory tests that measure how well blood is clotting and create an inaccurate result. Patients should speak with their healthcare provider about how this may affect their care. Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. Patients should only use Hemlibra for the condition it was prescribed. Patients should not give Hemlibra to other people, even if they have the same symptoms that they have. It may harm them. Patients should tell their healthcare provider about all the medicines they take, including prescription medicines, over-the-counter medicines, vitamins, or herbal supplements. Patients should keep a list of them to show their healthcare provider and pharmacist. Before using Hemlibra, patients should tell their healthcare provider about all of their medical conditions, including if they are pregnant, plan to become pregnant, are breastfeeding, or plan to breastfeed. Since Hemlibra was tested in males, there is no information on whether Hemlibra may impact an unborn baby or breast milk. Females who are able to become pregnant should use birth control during treatment. Side effects may be reported to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch . Side effects may also be reported to Genentech at (888) 835-2555. Please see Important Safety Information, including Serious Side Effects, as well as the Hemlibra full Prescribing Information and Medication Guide . About hemophilia A Hemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Hemophilia affects around 20,000 people in the United States, with hemophilia A being the most common form and approximately 50-60% of people living with a severe form of the disorder. People with hemophilia A either lack or do not have enough of a clotting protein called factor VIII. In a healthy person, when a bleed occurs, factor VIII brings together the clotting factors IXa and X, which is a critical step in the formation of a blood clot to help stop bleeding. Depending on the severity of their disorder, people with hemophilia A can bleed frequently, especially into their joints or muscles. These bleeds can present a significant health concern as they often cause pain and can lead to chronic swelling, deformity, reduced mobility and long-term joint damage. A serious complication of treatment is the development of inhibitors to factor VIII replacement therapies. Inhibitors are antibodies developed by the body’s immune system that bind to and block the efficacy of replacement factor VIII, making it difficult, if not impossible, to obtain a level of factor VIII sufficient to control bleeding. About Genentech in hemophilia In 1984, Genentech scientists were the first to clone recombinant factor VIII in response to the contaminated hemophilia blood supply crisis of the early 1980s. For more than 20 years, Genentech has been developing medicines to bring innovative treatment options to people with diseases of the blood within oncology, and in hemophilia A. Genentech is committed to improving treatment and care in the hemophilia community by delivering meaningful science and clinical expertise. For more information visit http://www.gene.com/hemophilia . About Genentech Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com .

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Indication	Country/Location	Organization	Date
Hemophilia A	US	Takeda Pharmaceuticals U.S.A., Inc.	21 Dec 1979
Hemophilia A	US	Baxter Healthcare Corp.	21 Dec 1979
Hemophilia B	US	Takeda Pharmaceuticals U.S.A., Inc.	21 Dec 1979
Hemophilia B	US	Baxter Healthcare Corp.	21 Dec 1979

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02764489 (CTgov)	Phase 3	Hemophilia B \| Hemophilia A	45	FEIBA (Part 1:FEIBA 85 ± 15 U/kg Regular Volume at 2 U/kg/Min Rate)	krmfclcwsh(pkzexhcwid) = xlqxgdznco clxzkrztwx (syodgsyktg, nyqsaeqmdd - chwcrwwgrk) View more	-	09 Feb 2023
				FEIBA (Part 1:FEIBA 85±15 U/kg 50% Reduced Volume at 2 U/kg/Min Rate)	krmfclcwsh(pkzexhcwid) = rltcyqdjtr clxzkrztwx (syodgsyktg, rjbqbwpoae - vaexlhdhdt) View more
ISTH2020	Not Applicable	Hemorrhage	-	Activated Prothrombin Complex Concentrates (aPCCs)	buyttrxpje(lwdkrkmlmy) = xniewzkagv dhktuygpcu (jcluimjeua ) View more	-	12 Jul 2020
P5.055 (AAN2017)	Not Applicable	Intracranial Hemorrhages	-	Weight-based activated prothrombin complex concentrate (aPCC)	cdsrnclwul(wljctnwuio) = ofuijzzngm fbxnhrqffp (fneqlmafwt ) View more	Positive	18 Apr 2017
				Fresh frozen plasma (FFP)	cdsrnclwul(wljctnwuio) = hhyitkbegg fbxnhrqffp (fneqlmafwt ) View more

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