A Multi-centre, Open-label, Single-arm, Non-interventional Post-marketing Study to Investigate Safety and Clinical Parameters of Alhemo® Under Routine Clinical Practice in Japan

The purpose of the study is to investigate the safety and effectiveness of Alhemo® in all participants under real-world clinical practice in Japan. Participants will get Alhemo® as prescribed by the study doctor. The study will last for about 2 years.

Start Date08 Aug 2024

Sponsor / Collaborator

Novo Nordisk A/S

NCT05135559 / RecruitingPhase 3

Open-label Study Investigating Efficacy, Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors

This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use.
Participants will have to inject the study medicine every day under the skin with a pen-injector.
The study will last for at least 2 years and up to about 4 years. The length of time the participant will be in the study depends on if the study medicine will be available for purchase in their country.

Start Date24 Mar 2022

Sponsor / Collaborator

Novo Nordisk A/S

NCT04082429 / Active, not recruitingPhase 3

Efficacy and Safety of Concizumab Prophylaxis in Patients With Haemophilia A or B Without Inhibitors

This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group participants will get study medicine from the start of the study. In the other group participants will continue with their normal medicine and get study medicine after 6 months. Which treatment the participant gets is decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will have to inject themselves with the study medicine 1 time every day under the skin. This can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for up to 6.5 years. The length of time the participant will be in the study depends on when they agreed to take part or when the medicine is available for purchase in their country (21 April 2026 at the latest). Participants will have to come to the clinic for up to 40 times. The time between visits will be approximately 4 weeks for the first 6 to 12 months depending on the group participants are in, and approximately 8 weeks for the rest of the study. If the participant attends extra visits due to the prescription medicine not being available for purchase in their country, these will be 14 weeks apart. Participants will be asked to record information in an electronic diary during the study and may also be asked to wear an activity tracker.

Start Date13 Nov 2019

Sponsor / Collaborator

Novo Nordisk A/S

100 Clinical Results associated with Concizumab-mtci

100 Translational Medicine associated with Concizumab-mtci

100 Patents (Medical) associated with Concizumab-mtci

Literatures (Medical) associated with Concizumab-mtci

31 Dec 2024·mAbs

Antibodies to watch in 2024

Article

Author: Crescioli, Silvia ; Kaplon, Hélène ; Wang, Lin ; Reichert, Janice M. ; Visweswaraiah, Jyothsna ; Chenoweth, Alicia

The 'Antibodies to Watch' article series provides an annual summary of commercially sponsored monoclonal antibody therapeutics currently in late-stage clinical development, regulatory review, and those recently granted a first approval in any country. In this installment, we discuss key details for 16 antibody therapeutics granted a first approval in 2023, as of November 17 (lecanemab (Leqembi), rozanolixizumab (RYSTIGGO), pozelimab (VEOPOZ), mirikizumab (Omvoh), talquetamab (Talvey), elranatamab (Elrexfio), epcoritamab (EPKINLY), glofitamab (COLUMVI), retifanlimab (Zynyz), concizumab (Alhemo), lebrikizumab (EBGLYSS), tafolecimab (SINTBILO), narlumosbart (Jinlitai), zuberitamab (Enrexib), adebrelimab (Arelili), and divozilimab (Ivlizi)). We briefly review 26 product candidates for which marketing applications are under consideration in at least one country or region, and 23 investigational antibody therapeutics that are forecast to enter regulatory review by the end of 2024 based on company disclosures. These nearly 50 product candidates include numerous innovative bispecific antibodies, such as odronextamab, ivonescimab, linvoseltamab, zenocutuzumab, and erfonrilimab, and antibody-drug conjugates, such as trastuzumab botidotin, patritumab deruxtecan, datopotamab deruxtecan, and MRG002, as well as a mixture of two immunocytokines (bifikafusp alfa and onfekafusp alfa). We also discuss clinical phase transition and overall approval success rates for antibody therapeutics, which are crucial to the biopharmaceutical industry because these rates inform decisions about resource allocation. Our analyses indicate that these molecules have approval success rates in the range of 14-32%, with higher rates associated with antibodies developed for non-cancer indications. Overall, our data suggest that antibody therapeutic development efforts by the biopharmaceutical industry are robust and increasingly successful.

01 Dec 2024·Lancet Haematology

Concizumab prophylaxis in people with haemophilia A or haemophilia B without inhibitors (explorer8): a prospective, multicentre, open-label, randomised, phase 3a trial

Article

Author: Eichler, Hermann ; Tran, Huyen ; Høgh Nielsen, Amalie Rhode ; Šaulytė Trakymienė, Sonata ; Villarreal Martinez, Laura ; Benson, Gary ; Apte, Shashikant ; Jiménez Yuste, Victor ; Young, Guy ; Sutton, Christopher ; Chan, Anthony K C ; Windyga, Jerzy ; Chowdary, Pratima ; Thaung Zaw, Jay Jay ; Sathar, Jameela ; Wheeler, Allison P ; Angchaisuksiri, Pantep ; Matsushita, Tadashi ; Astermark, Jan

BACKGROUND:

Concizumab is an anti-tissue factor pathway inhibitor monoclonal antibody in development as a once-daily, subcutaneous prophylaxis for patients with haemophilia A or haemophilia B with or without inhibitors. We aimed to assess the efficacy and safety of concizumab in patients with haemophilia A or B without inhibitors. Here we report the results from the confirmatory analysis cutoff.

METHODS:

This prospective, multicentre, open-label, randomised, phase 3a trial (explorer8) was conducted at 69 investigational sites in 31 countries. Eligible patients were male, aged 12 years or older, and had congenital severe haemophilia A or moderate or severe haemophilia B without inhibitors and with documented treatment with clotting factor concentrate in the 24 weeks before screening. The trial was paused because of non-fatal thromboembolic events in three patients (two from this trial [explorer8] and one from a related trial in haemophilia with inhibitors [explorer7; NCT04083781]) and restarted with mitigation measures, including a revised dosing regimen of subcutaneous concizumab at 1·0 mg/kg loading dose on day 1 and subsequent daily doses of 0·20 mg/kg from day 2, with options to decrease to 0·15 mg/kg, stay on 0·20 mg/kg, or increase to 0·25 mg/kg on the basis of concizumab plasma concentration measured after 4 weeks on concizumab. Patients recruited after treatment restart were randomly assigned 1:2 using an interactive web response system to receive no prophylaxis and continue on-demand clotting factor (group 1) or concizumab prophylaxis (group 2). The primary endpoints were the number of treated spontaneous and traumatic bleeding episodes for patients with haemophilia A and haemophilia B separately, assessed at the confirmatory analysis cutoff in randomly assigned patients. Analyses were by intention-to-treat. There were two additional groups containing non-randomly-assigned patients: group 3 contained patients who entered the trial before the trial pause and were receiving concizumab in the phase 2 trial (explorer5; NCT03196297), and group 4 contained patients who received previous clotting factor concentrate prophylaxis or on-demand treatment in the non-interventional trial (explorer6; NCT03741881), patients randomly assigned to groups 1 or 2 before the treatment pause, and patients from explorer5 enrolled after the treatment pause. The safety analysis set contained all patients who received concizumab. Superiority of concizumab over no prophylaxis was established if the two-sided 95% CI of the treatment ratio was less than 1 for haemophilia A and for haemophilia B. This trial is registered with ClinicalTrials.gov, NCT04082429, and its extension part is ongoing.

FINDINGS:

Patients were recruited between Nov 13, 2019 and Nov 30, 2021; the cutoff date for the analyses presented was July 12, 2022. 173 patients were screened, of whom 148 (86%) were randomly assigned or allocated to the four groups in the study after trial restart on Sept 30, 2020 (nine with haemophilia A and 12 with haemophilia B in group 1; 18 with haemophilia A and 24 with haemophilia B in group 2; nine with haemophilia A in group 3; and 46 with haemophilia A and 30 with haemophilia B in group 4). The estimated mean annualised bleeding rate ratio for treated spontaneous and traumatic bleeding episodes during concizumab prophylaxis versus no prophylaxis was 0·14 (95% CI 0·07-0·29; p<0·0001) for patients with haemophilia A and 0·21 (0·10-0·45; p<0·0001) for patients with haemophilia B. The most frequent adverse events in patients who received concizumab were SARS-CoV-2 infection (19 [13%] of 151 patients), an increase in fibrin D-dimers (12 [8%] patients), and upper respiratory tract infection (ten [7%] patients). There was one fatal adverse event possibly related to treatment (intra-abdominal haemorrhage in a patient from group 4 with haemophilia A with a long-standing history of hypertension). No thromboembolic events were reported between the trial restart and confirmatory analysis cutoff.

INTERPRETATION:

Concizumab was effective in reducing the bleeding rate compared with no prophylaxis and was considered safe in patients with haemophilia A or B without inhibitors. The results of this trial suggest that concizumab has the potential to be one of the first subcutaneous treatment options for patients with haemophilia B without inhibitors.

FUNDING:

Novo Nordisk.

01 Nov 2024·EUROPEAN JOURNAL OF HAEMATOLOGY

Disease and treatment burden of patients with haemophilia entering the explorer6 non‐interventional study

Article

Author: Frei‐Jones, Melissa ; Brown Frandsen, Renée ; Windyga, Jerzy ; Apte, Shashikant ; Villarreal Martinez, Laura ; Lyu, Chuhl Joo ; Zozulya, Nadezhda ; Mahlangu, Johnny ; Fujii, Teruhisa ; Neergaard, Jesper Skov ; Sathar, Jameela ; Thaung Zaw, Jay Jay ; Stasyshyn, Oleksandra ; Tran, Huyen

Abstract:

Objectives:

We aimed to characterise baseline disease and treatment burden in a large population with haemophilia A/B, both with (HAwI/HBwI) and without (HA/HB) inhibitors.

Methods:

The prospective, non‐interventional explorer6 study included patients ≥12 years old with severe HA, severe/moderate HB or HAwI/HBwI of any severity, treated according to local standard of care (excluding previous/current exposure to concizumab or emicizumab). Baseline characteristics and historical clinical data were collected and patient‐reported outcomes, including treatment burden, were assessed.

Results:

The explorer6 study enrolled 231 patients with haemophilia (84 HAwI/HBwI) from 33 countries. At baseline, patients with HA/HB treated with prophylaxis had the lowest median annualised bleeding rates (ABRs; 2.0), irrespective of haemophilia type; of these patients, 27.5% (HA) and 31.4% (HB) had target joints. Patients with HAwI/HBwI treated episodically reported the highest treatment burden. Of these patients, 28.5% (HAwI) and 25.1% (HBwI) performed sports activities in the month before screening.

Conclusion:

Despite receiving routine clinical care, historical and baseline information from patients enrolled in explorer6 showed that patients with HA/HB treated episodically and patients with HAwI/HBwI had higher ABRs, higher treatment burden and participated in sports less than those with HA/HB treated with prophylaxis. Emerging treatments could be beneficial in addressing these unmet medical needs.

News (Medical) associated with Concizumab-mtci

21 Dec 2024

·www.prnewswire.com

FDA approves Alhemo® injection as once-daily prophylactic treatment to prevent or reduce the frequency of bleeding episodes for adults and children 12 years of age and older with hemophilia A or B with inhibitors

FDA approval is based on data from the pivotal phase 3 trial (explorer7) establishing the safety and efficacy of Alhemo® for daily prevention of bleeds in adults and pediatric patients 12 years of age and older living with hemophilia A or B with inhibitors Results from the pivotal trial showed an 86% reduction in treated spontaneous and traumatic bleeds in patients using Alhemo® prophylaxis compared to no prophylaxis1 This approval marks the first subcutaneous injection treatment of its kind for use in this patient population PLAINSBORO, N.J., Dec. 20, 2024 /PRNewswire/ -- Novo Nordisk announced today that the U.S. Food and Drug Administration (FDA) approved Alhemo® (concizumab-mtci) injection as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B with inhibitors, continuing its more than 35-year commitment to those living with rare bleeding disorders.1,2 Alhemo® is a tissue factor pathway inhibitor (TFPI) antagonist that is dosed in a prefilled, premixed pen for subcutaneous injection (60 mg/1.5 mL, 150 mg/1.5 mL, or 300 mg/3 mL) via a thin 32 gauge, 4 mm needle, which is provided separately.1,3 Currently, many treatments for hemophilia A or B with inhibitors are administered via intravenous infusions, and Alhemo® is the first subcutaneous injection treatment of its kind for this population.1,4,5 Alhemo® is designed to block a protein called TFPI in the body that stops blood from clotting. By blocking TFPI, Alhemo® improves the production of thrombin, a protein that helps to clot the blood and prevent bleeding, when the other clotting factors are missing or deficient in the presence of inhibitors.3,5 An estimated 30% of patients living with severe hemophilia A and 5-10% of those with severe hemophilia B develop inhibitors, which makes treatment of hemophilia in some patients significantly more challenging.4,6 While treatments have improved the lives of many living with hemophilia, those with hemophilia B with inhibitors still experience a disease and treatment burden due to limited prophylactic treatment options to prevent bleeding.4 Because of the unmet medical needs in this population, and based on the Phase 2 clinical trial results, the FDA granted Breakthrough Therapy designation for Alhemo® in hemophilia B with inhibitors.3 "The approval of Alhemo® signifies a remarkable achievement in prophylactic hemophilia treatment for individuals with inhibitors aged 12 years and older who, in some cases, currently have few options," said Anna Windle, SVP Clinical Development, Novo Nordisk. "As the first treatment of its kind for this population, Alhemo® represents a significant step in helping to address the unmet needs of patients with hemophilia with inhibitors, highlighting Novo Nordisk's commitment to patient-centric innovations in rare diseases." The primary objective from the pivotal Phase 3 explorer7 study compared the number of treated spontaneous and traumatic bleeding episodes, as measured by annual bleeding rate (ABR), showed an 86% reduction of ABR in patients randomized to receive Alhemo® prophylaxis compared to no prophylaxis (ABR ratio of 0.14, 95% confidence interval [CI], 0.07 to 0.29, p-value <0.001). The estimated mean ABR was 1.7 for patients on Alhemo® prophylaxis compared to 11.8 for patients with no prophylaxis and the overall median ABR was zero for treated spontaneous and traumatic bleeds compared with 9.8 ABR in patients with no prophylaxis.5 As a supportive secondary efficacy endpoint, 64% of the patients randomized to receive Alhemo® prophylaxis treatment experienced zero treated spontaneous and traumatic bleeds during the first 24 weeks of treatment vs. 11% with no prophylaxis.5 In the explorer7 study, the most common adverse reactions reported in ≥5% of patients randomized to receive Alhemo® were injection site reactions (18%) and urticaria (6%). Serious adverse reactions were renal infarct and hypersensitivity reaction.1 "The development of inhibitors remains the most serious treatment-related complication for people living with hemophilia. For patients with inhibitors, especially in hemophilia B, their hemophilia may remain poorly controlled and pose a life-threatening risk," said Amy Shapiro, MD, CEO and co-medical director at the Indiana Hemophilia & Thrombosis Center, Inc. "The approval of Alhemo® – a first-of-its-kind, prophylaxis, subcutaneous injection pen for adults and children 12 years and older with hemophilia A and B with inhibitors – provides a much-needed alternative to the current standard of care in hemophilia B with inhibitors, while offering patients with hemophilia A with inhibitors more treatment options, ultimately providing more patients with inhibitors the opportunity to personalize their care and address current treatment gaps." In addition to the U.S., Alhemo® is currently approved in Australia, Japan, Switzerland and the EU, with specific indications varying by country. About the explorer7 study Explorer7 is a clinical trial that established the efficacy and safety of Alhemo® for adults and pediatric patients 12 years of age and older living with hemophilia A or B with inhibitors.1,5 In explorer7, 52 males were randomly assigned in a 1:2 ratio to receive no prophylaxis (arm 1, n=19), or Alhemo® prophylaxis (arm 2, n=33) and 81 males were nonrandomly assigned to receive Alhemo® prophylaxis (arms 3 and 4).1,5 The initial loading dose of Alhemo® was 1 mg per kilogram of body weight, followed by 0.2 mg per kilogram daily, and potentially individualized on the basis of concizumab-mtci plasma concentration as measured at week 4.1,5 The primary analysis was carried out when all patients in arms 1 and 2 completed at least 24 or 32 weeks, respectively, and compared the number of treated spontaneous and traumatic bleeding episodes, measured as ABR, between arms one and two.1,5 Supportive secondary endpoints, such as percent of patients experiencing zero bleeds, are reported as descriptive results only.5 About hemophilia with inhibitors Hemophilia is a rare bleeding disorder that affects approximately 800,000 people worldwide and 32,000 people in the US, that impairs the body's ability to make blood clots, a process needed to stop bleeding.7-9 It is caused by a mutation in a gene that provides instructions for making the clotting factor proteins needed to form a blood clot.9 This change can prevent the clotting protein from working properly or be missing altogether.9 There are different types of hemophilia, which are characterized by the type of clotting factor protein that is defective or missing. Hemophilia A is caused by low levels of clotting factor VIII (FVIII), while hemophilia B is caused by low levels of clotting factor IX (FIX).9 Hemophilia is often treated by replacing the missing clotting factor via intravenous infusions, also known as replacement therapy.9 However, sometimes the body can produce inhibitors as an immune response to the clotting factors in the therapy, which means replacement therapy does not work and limits overall treatment options.9,10 About Alhemo® (concizumab-mtci) injection Alhemo® is a tissue factor pathway inhibitor (TFPI) antagonist, a protein in the body that helps to stop blood from clotting. By inhibiting TFPI, Alhemo® enhances factor Xa (FXa) production during the initiation phase of coagulation, leading to improved thrombin generation and clot formation in patients with hemophilia A or B with inhibitors. The effect of Alhemo is not influenced by the presence of inhibitory antibodies to FVIII or FIX and Alhemo does not induce or enhance the development of direct inhibitors to FVIII or FIX. Alhemo® is approved as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12 years of age and older with hemophilia A or B with inhibitors in the US.1 What is Alhemo®? Alhemo® (concizumab-mtci) injection 60 mg, 150 mg, or 300 mg is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children 12 years of age and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors. It is not known if Alhemo® is safe and effective in people receiving ongoing immune tolerance induction (ITI) It is not known if Alhemo® is safe and effective for hemophilia A and B with and without inhibitors in children younger than 12 years of age Important Safety Information What is the most important information I should know about Alhemo®? It is important to follow the daily dosing schedule of Alhemo® to stay protected against bleeding. This is especially important during the first 4 weeks of treatment to make sure a correct maintenance dose is established. Use Alhemo® exactly as prescribed by your healthcare provider (HCP). Do not stop using Alhemo® without talking to your HCP. If you miss doses, or stop using Alhemo®, you may no longer be protected against bleeding Your HCP may prescribe bypassing agents during treatment with Alhemo®. Carefully follow your HCP's instructions regarding when to use on-demand bypassing agents, and the recommended dose and schedule for breakthrough bleeds Do not use Alhemo® if you are allergic to concizumab-mtci or any of the ingredients in Alhemo®. Before using Alhemo®, tell your HCP about all of your medical conditions, including if you: Have a planned surgery. Your HCP may stop treatment with Alhemo® before your surgery. Talk to your HCP about when to stop using Alhemo® and when to start it again if you have a planned surgery. Are pregnant or plan to become pregnant. It is not known if Alhemo® may harm your unborn baby. Females who are able to become pregnant Your HCP may do a pregnancy test before you start treatment with Alhemo®. You should use an effective birth control (contraception) method during treatment with Alhemo® and for 7 weeks after ending treatment. Talk to your HCP about birth control methods that you can use during this time Are breastfeeding or plan to breastfeed. It is not known if Alhemo® passes into your breast milk. Talk to your HCP about the best way to feed your baby during treatment with Alhemo® Tell your HCP about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Know the medicines you take. Keep a list of them to show your HCP and pharmacist when you get a new medicine. How should I use Alhemo®? Change (rotate) your injection site with each injection. Do not use the same site for each injection To determine the right maintenance dose for you, your HCP will do a blood test to check the amount of Alhemo® in your blood. Your HCP may do additional blood tests during treatment with Alhemo® Do not share your Alhemo® pens and needles with another person, even if the needle has been changed. You may give another person an infection or get an infection from them If you miss a dose of Alhemo® during the first 4 weeks of treatment, contact your HCP right away. Your HCP will tell you how much Alhemo® to inject What are the possible side effects of Alhemo®? Alhemo® may cause serious side effects, including: Blood clots (thromboembolic events). Alhemo® may cause blood clots to form in blood vessels, such as in your arms, legs, heart, lung, brain, eyes, kidneys, or stomach. You may be at risk for getting blood clots during treatment with Alhemo® if you use high or frequent doses of factor products or bypassing agents to treat breakthrough bleeds, or if you have certain conditions. Get medical help right away if you have any signs and symptoms of blood clots, including: swelling, warmth, pain, or redness of the skin; headache; trouble speaking or moving; eye pain or swelling; sudden pain in your stomach or lower back area; feeling short of breath or severe chest pain; confusion; numbness in your face; and problems with your vision Allergic reactions. Alhemo® can cause allergic reactions, including redness of the skin, rash, hives, itching, and stomach-area (abdominal) pain. Stop using Alhemo® and get emergency medical help right away if you develop any signs or symptoms of a severe allergic reaction, including: itching on large areas of skin; trouble swallowing; wheezing; pale and cold skin; dizziness due to low blood pressure; redness or swelling of lips, tongue, face, or hands; shortness of breath; tightness of the chest; and fast heartbeat The most common side effects of Alhemo® include: bruising, redness, bleeding, or itching at the site of injection, and hives. Please click HERE for Alhemo® Prescribing Information and Medication Guide About Novo Nordisk Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases, built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 72,000 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com, Facebook, Instagram, X, LinkedIn, and YouTube. References Alhemo® (concizumab-mtci) injection, for subcutaneous use [package insert]. Plainsboro, NJ: Novo Nordisk Inc. Hedner U. History of rFVIIa therapy. Thromb Res. 2010;125 Suppl 1:S4-S6. doi:10.1016/j.thromres.2010.01.021 Shapiro AD. Concizumab: a novel anti-TFPI therapeutic for hemophilia. Blood Adv. 2021;5(1):279 Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition [published correction appears in Haemophilia. 2021 Jul;27(4):699]. Haemophilia. 2020;26 Suppl 6:1-158. doi:10.1111/hae.14046 Matsushita T, Shapiro A, Abraham A, et al. Phase 3 trial of concizumab in hemophilia with inhibitors. N Engl J Med. 2023; 389(9): 783-794. Male C, Andersson NG, Rafowicz A, et al. Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study. Haematologica. 2021 106(1):123-129. doi: 10.3324/haematol.2019.239160. PMID: 31919092; PMCID: PMC7776246. World Federation of Hemophilia. Annual Global Survey 2021. Accessed December 2024. Available at . Centers for Disease Control and Prevention (CDC). Factor VIII and Factor IX. Accessed December 2024. Available at . Centers for Disease Control and Prevention (CDC). About Hemophilia. Accessed December 2024. Available at . Kim JY, You CW. The prevalence and risk factors of inhibitor development of FVIII in previously treated patients with hemophilia A. Blood Res. 2019 Sep;54(3):204-209. doi: 10.5045/br.2019.54.3.204. Further information Media: [email protected] Ambre James-Brown +45 3079 9289 [email protected] Liz Skrbkova (US) +1 609 917 0632 (Mobile) [email protected] Alhemo® is a registered trademark of Novo Nordisk Health Care AG. Novo Nordisk is a registered trademark of Novo Nordisk A/S. © 2024 Novo Nordisk All rights reserved. US24AHM00077 December 2024 SOURCE Novo Nordisk WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultPhase 3Drug ApprovalBreakthrough TherapyPhase 2

17 Dec 2024

·www.pharmaceutical-technology.com

Novo Nordisk invests $1.2bn in new Denmark facility

Credit: Kittyfly/Shutterstock. Denmark-based pharma giant Novo Nordisk has announced plans to invest DKr8.5bn ($1.2bn) to establish a new production facility in Odense, Denmark. Announcing the news yesterday (16 December), the company said that the new site will include a production facility manufacturing multiple therapies for rare diseases, including haemophilia. It will also include a warehouse covering more than 40,000m². The company’s haemophilia portfolio includes Alhemo (concizumab) and its antibody asset Mim8, with the Phase III FRONTIER2 trial (NCT05053139) in haemophilia A completing earlier this year. Novo Nordisk has not yet published data from the study. Novo Nordisk is also running a Phase I trial (NCT06649630) of its oral antibody, Inno8, for haemophilia. This announement comes just days after Novo Holdings, the controlling shareholder of Novo Nordisk, closed a $16.5bn deal to acquire Catalent , a global contract development and manufacturing organisation. The deal was greenlighted by the Federal Trade Commission and the European Commission. It will boost the manufacturing capability for its glucagon-like peptide-1 receptor agonists (GLP-1RAs) Wegovy and Ozempic, which have suffered global shortages. Construction work on the site has already commenced and Novo Nordisk expects the facilities to be completed in 2027. Up to 1,000 external employees will be involved in the construction, and the site will produce 400 new and permanent jobs. Speaking on the new Denmark facility, Henrik Wulff, executive vice-president of product supply, quality and IT of Novo Nordisk, said: “The facility will utilise advanced technology and innovative equipment to ensure the highest quality to patients and meet the growing global demand for our life-changing medicines. “We are proud to build on our heritage in Denmark and look forward to embarking on this journey in Odense, a well-connected city with a dynamic community and talented workforce,” he added. Novo Nordisk also plans to plant 4,000 new trees at the site in an effort to preserve local nature. The new facility buildings will also be equipped with solar panels for on-site electricity generation, whilst the local landscape will be reconstructed using excess soil, wooden materials and other sustainable resources to create lakes, forests and community spaces open to the public. Novo Nordisk is Europe’s most valuable listed company, having found huge success with its weight-loss and type 2 diabetes injections Wegovy and Ozempic. The company expects to significantly increase production this year, announcing in June that it expects to invest $6.8bn this year, up from $3.9bn in 2023.

AcquisitionPhase 1Phase 3Phase 2

16 Dec 2024

·endpts.com

Novo Nordisk to spend $1.2B for new rare disease drug factory in Denmark

Just days after winning FTC approval to take on three of Catalent’s facilities, Novo Nordisk said it is investing DKK 8.5 billion ($1.2 billion) to build a new factory in Denmark to make its rare disease assets. The new 40,000 square-meter build in Odense will manufacture different products, including ones for hemophilia, Novo said in a Monday release. According to Novo, this is the first time in a century it is breaking ground for a new site in its home country. Around 400 new jobs will be created and the factory will be completed in 2027. As a part of its ESG initiative, Novo will install solar panels, plant more than 4,000 new trees around the site, and have landscaping with locals in mind. The Danish pharma’s hemophilia portfolio includes concizumab. The EMA gave a positive opinion for this candidate for hemophilia A and B in October, a comeback for the drug after the FDA issued a CRL in May 2023. Novo also has a Phase 3 antibody asset called Mim8 under investigation for hemophilia A, with plans to submit the drug for regulatory approval by the end of the year. Novo is also studying a Phase 1 oral antibody for hemophilia. The company has other rare disease assets in its pipeline, including a Phase 3 small molecule being studied for sickle cell disease.

Phase 3Phase 1

100 Deals associated with Concizumab-mtci

External Link

KEGG	Wiki	ATC	Drug Bank
D11847	-	-	DB12820

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Hemophilia A	AU	Novo Nordisk Pharmaceuticals Pty Ltd.	05 Jul 2023
Hemophilia B	CA	Novo Nordisk Canada, Inc.	10 Mar 2023

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Hemophilia	Phase 3	US	Novo Nordisk A/S	13 Nov 2019
Hemophilia	Phase 3	JP	Novo Nordisk A/S	13 Nov 2019
Hemophilia	Phase 3	AU	Novo Nordisk A/S	13 Nov 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


explorer7 and explorer8 (ASH2023)	Phase 3	Hemophilia	278	Concizumab prophylaxis	pmpmyfzugn(ngjsuncpuw) = eteusdsuky ptdhphkbuq (ctenieycnb )	-	09 Dec 2023
ISTH2023	Phase 3	-	-	Concizumab	htqiqqumvi(gpmnhgdcjr) = ywtgtmvfzn drldhgysnw (kvqzafwvfb )	-	24 Jun 2023
				Concizumab	htqiqqumvi(gpmnhgdcjr) = ylhlqwuqtb drldhgysnw (kvqzafwvfb, 21)
ISTH2023	Not Applicable	Hemophilia A	80	Concizumab pen-injector	yexjyafioh(tzjrsfeoxk) = kjkiflhbay tmysrwxeui (wmntsroeix, 91 - 100) View more	-	24 Jun 2023
NCT04083781 (explorer7, ASH 12022 \| ASH 22022) Manual	Phase 3	Hemophilia B \| Hemophilia A	25	Concizumab (Concizumab prophylaxis)	ansexyjipp(tmiwkohklm) = ttoqybuuwx wllovpjjdc (eyzbeqcgru, -23.7 to -10.3)	Positive	15 Nov 2022
				Concizumab (non-concizumab prophylaxis treatment)	ansexyjipp(tmiwkohklm) = lsgiqmizjq wllovpjjdc (eyzbeqcgru, -9.4 to 15.3)
NCT04083781 (explorer7, GlobeNewswire) Manual	Phase 3	Hemophilia B	133	Concizumab	ibhmhbqodu(fmkjwccodt) = gkelvstnoa ldnfpdtulv (ersgsdsswl ) View more	Positive	10 Jul 2022
				no prophylaxis	sjkjqwzdfj(tlohzreqqr) = rwtpsobsrn urkkczafxe (tspaxkhkvs )
NCT03196284 \| NCT03196297 (explore5 \| explore4, Pubmed \| Blood Adv) Manual	Phase 2	Hemophilia B	-	concizumab (explore4)	plgsiohjxr(uepqcffulr) = qtlnyqzuwy rfuopkmjzl (ilwhutwxoe, 3.2 - 7.2) View more	Positive	15 Mar 2022
				concizumab (explore5)	plgsiohjxr(uepqcffulr) = qawfmuajch rfuopkmjzl (ilwhutwxoe, 4.1 - 9.9) View more
NCT02490787 (Explorer™3, Pubmed \| J Thromb Haemost) Manual	Phase 1	Hemophilia A	24	concizumab	bvrvtmhjqv(uebfqwjofx) = No serious adverse events ldsvievstv (nogkjtqnte )	Positive	01 Nov 2018
				Placebo

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis