PALETTE- Adaptive Platform Trial for Personnalisation of Sepsis Treatment in Children and Adults: a Multi-national, Treatable Traits-guided, Adaptive, Bayesian Basket Trial"

PALETTE is a perpetual adaptive platform to efficiently study sepsis interventions within 'treatable traits' in all-ages patients enabling prompt evaluation of pandemic treatments. Treatable traits, therapeutic targets identified by phenotypes or endotypes (defined by biological mechanism or by treatment response) through validated biomarkers (measurable characteristic reflecting normal or pathogenic processes, or treatment responses), may include multi-omics, cellular, immune, metabolic, endocrine features, or intelligent algorithms. PALETTE Bayesian adaptive design enables parallel investigations of multiple interventions for sepsis, and quick inclusion of pandemic pathogens. PALETTE's new conceptual model will respond to the challenges of standard approaches, i.e. series of sepsis trials, each investigating one or two interventions, expensive, time consuming, and inappropriate in pandemic context.

Start Date01 Apr 2026

Sponsor / Collaborator

Assistance Publique des Hôpitaux de Paris SA

NCT06666335

/ Not yet recruitingPhase 4

A Prospective, Open-label, Single Arm, Multicenter, Post-authorization Efficacy and Safety Study of Subcutaneous Anakinra in Chinese Patients With Colchicine-resistant Familial Mediterranean Fever (FMF)

The purpose of this study is to evaluate the efficacy and safety of anakinra in Chinese patients with colchicine-resistand Familial Mediterranian Fever (FMF). The study consists of up to one month screening, to see if a patient is suitable to the study, 6 months of treatment with anakinra and one month safety follow up after last dose of anakinra. In total 3 patients, male and female from 2 years of age (minimum 10kg weight), will be enrolled to the study.

Start Date01 Sep 2025

Sponsor / Collaborator

Swedish Orphan Biovitrum AB

[+1]

NCT06703216

/ Not yet recruitingPhase 1/2IIT

Pilot Study of Pre-emptive Anakinra for the Prevention of Severe Cytokine Release Syndrome in Children and Young Adults With B-Acute Lymphoblastic Leukemia Receiving Chimeric Antigen Receptor (CAR) T Cells

Objectives: The primary objective of this study will be to evaluate the impact of pre-emptive use of anakinra on the rate of severe cytokine release syndrome (CRS) following CD19-directed chimeric antigen receptor (CAR) T-cell therapy for B-acute lymphoblastic leukemia (B-ALL) in children and young adults.
Patient Population: Children and young adults <25 years of age undergoing CAR T-cell therapy for B-ALL with bone marrow disease burden of ≥5% involvement or detectable peripheral blasts within 2 weeks of the initiation of lymphodepleting chemotherapy.
Study Design: This is a pilot single arm study. The investigators will inquire into the efficacy and safety of using anakinra pre-emptively to reduce the rate of severe CRS in patients with >/=5% bone marrow blasts or lymphoblasts in the peripheral blood.
Treatment Plan:
This is a single arm unblinded study in which patients will receive anakinra, 2.5 mg/kg (max 100mg), IV every 12 hours starting at the onset of persistent fever (fever >38.5⁰ C x 2 occurrences separated by at least 4 hours in a 24 hour period). If there is persistence or progression of CRS, anakinra frequency will be increased to 2.5mg/kg IV (max 100mg), every 6 hours. Anakinra will be continued until 48 hours after resolution of CRS and ICANS, and at least 7 days post-CAR T infusion. If dose and frequency of anakinra is increased, the increased dose of anakinra will be continued until 48 hours after resolution of CRS and immune effector cell-associated neurotoxicity syndrome (ICANS) and at least 7 days post-CAR T infusion. For CRS worsening beyond dose escalation of anakinra, CRS will be managed as per standard of care management. Participants will be followed for 12 months following enrollment in the study and disease evaluations will be performed as per routine clinical care following CAR T-cell therapy.

Start Date01 Aug 2025

Sponsor / Collaborator

Ann & Robert H. Lurie Children's Hospital of Chicago

[+1]

100 Clinical Results associated with Anakinra (Amgen)

100 Translational Medicine associated with Anakinra (Amgen)

100 Patents (Medical) associated with Anakinra (Amgen)

2,756

Literatures (Medical) associated with Anakinra (Amgen)

31 Dec 2025·ANNALS OF MEDICINE

Risk of serious bacterial infections in inflammatory rheumatic or bowel disease patients during biological therapies: nationwide Danish cohort study

Article

Author: Østergaard, Anne Ahrens ; Platz, Emma Cathrine Højberg ; Johansen, Isik Somuncu ; Byg, Keld-Erik ; Petersen, Inge ; Laugesen, Gustav Emil

INTRODUCTION:

To estimate the incidence rate ratio (IRR) of serious bacterial infections (SI) in patients with inflammatory bowel disease (IBD) or inflammatory rheumatic disease (IRD) before and after initiating biological therapy, and to assess comorbidities, infection sites, and the type of biological therapy.

METHODS:

This nationwide retrospective registry-based study used a cohort-crossover design to assess the relative risk of SI one year before and after treatment. Data from the Danish National Patient Registry included 20,216 patients with IBD or IRD. Diagnosis codes for hospital contacts (inpatients, outpatients, day patients, and emergency department patients) and hospital-treated bacterial infections from 1994 to 2018 were linked using the Civil Registration System.

RESULTS:

There were 1235 infections during 19,529 person-years one year before treatment, compared to 1251 infections over 17,831 person-years one year after treatment initiation (IRR = 1.11, 95% CI [1.03-1.19], p = 0.01). The incidence rate was 2-fold higher in the first three months post-treatment compared to over one year. Increased risks were found for bacterial infections without specified site; ear, nose, and throat; and respiratory tract infections. Higher risks of SI after treatment initiation were associated with comorbidities, treatment initiation between 2015 and 2018, age ≥61 at inflammatory disease diagnosis [HR = 1.50 (1.31-1.72)], female sex, and corticosteroid co-administration. Anakinra showed a significantly increased risk of infection [HR = 2.02, 95%CI 1.03-3.97)] in IRD patients.

CONCLUSIONS:

We found a modestly increased risk of SI in the year following therapy initiation, with the highest risk in the first three months. This risk was associated with certain baseline factors, highlighting the need for thorough risk assessment when starting treatment.

01 Dec 2025·Current Pain and Headache Reports

Familial Mediterranean Fever (FMF): Emerging Concepts in Diagnosis, Pain Management, and Novel Treatment Options: A Narrative Review

Review

Author: Baptiste, Carlo Jean ; Norwood, Haley A ; Islam, Kazi N ; Nguyen, Ivan D ; Islam, Rahib K ; Ahmadzadeh, Shahab ; Kaye, Alan D ; Barrie, Sonnah ; McGregor, David W ; Robin, Connor J ; Parker-Actlis, Tomasina Q ; Shekoohi, Sahar

PURPOSE OF REVIEW:

Familial Mediterranean Fever (FMF) is a hereditary autoinflammatory disorder predominantly affecting individuals of Mediterranean and Middle Eastern descent, including those with certain heritages including Sephardic Jewish, Armenian, Turkish, and Arab. The disorder affects up to 1 in 200 people making it a very common etiology for pain states worldwide, including serositis mediated painful states of the chest, joint, and abdomen.

RECENT FINDINGS:

Defined by recurrent episodes of fever and inflammation, FMF can lead to not only severe pain, but complications such as renal amyloidosis, if untreated. This narrative review explores the genetic basis, clinical manifestations, diagnostic criteria, and current management strategies for FMF. Mutations in the MEFV gene result in the dysregulation of the pyrin inflammasome, leading to excessive production of inflammatory cytokines. Diagnosis primarily relies on clinical criteria supported by genetic testing. Colchicine remains the cornerstone of treatment, effectively preventing inflammatory attacks and complications. For colchicine-resistant patients, IL-1 antagonists like anakinra and canakinumab show promise, although their long-term benefits require further investigation. The present investigation underscores the importance of early diagnosis and integrated treatment approaches to improve patient outcomes, pain management, and quality of life.

01 Dec 2025·Current Cardiology Reports

The Role of the Advanced Practice Provider in a Pericardial Center of Excellence

Review

Author: Wensink, Emma ; Klein, Allan ; Agrawal, Ankit ; Heine, Mary ; Wang, Tom Kai Ming

Abstract:

Purpose of Review:

Pericarditis can be a chronic and complex disease requiring years of treatment. These patients require close monitoring of labs, medications and their side effects, as well as virtual and inpatient follow up. Due to the complexity of this disease, many of these patients are treated for months and sometimes years. Our review highlights the role Advanced practice providers (APP) play in managing the complexity of these patients by providing efficient and quality care.

Recent Findings:

Multi-modality cardiac imaging is the cornerstone to the evaluation and treatment of pericardial diseases. The addition of Interleukin (IL-1) blockers or biologics (Rilonacept, Anakinra) in the last few years provides targeted therapy for these patients. Using imaging guided therapy (IGT) these complex patients require close, continuous follow up and monitoring as well as frequent medication titration.

Summary:

The outcomes for these pericardial patients are improved with these centers due to the specialized medical and surgical care. Advanced Practice Providers play a vital role in a pericardial center with ordering the appropriate imaging and labs, handling medication titration, and providing patient education and continuity of care for these patients. They have been shown to decrease mortality, increase quality of care, and increase medication adherence.

News (Medical) associated with Anakinra (Amgen)

22 May 2025

·www.prnewswire.com

Celltrion's YUFLYMA® (adalimumab-aaty) receives FDA interchangeability designation for all its approved dosage forms and strengths

YUFLYMA ® (adalimumab-aaty) is a high-concentration (100mg/mL) and citrate-free formulation of Humira ® (adalimumab) biosimilar[1] Celltrion previously received interchangeability (IC) designation for YUFLYMA® (adalimumab-aaty) in prefilled syringes (20mg & 80mg); Expanded interchangeability (IC) designation applies to prefilled syringe (40mg) and autoinjectors (40mg and 80mg) INCHEON, South Korea, May 21, 2025 /PRNewswire/ -- Celltrion, Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted an expanded interchangeable designation for YUFLYMA ® (adalimumab-aaty), now including prefilled syringe (40mg) and autoinjectors (40mg and 80mg) presentations. With this approval, YUFLYMA is now fully interchangeable with the reference product, Humira ® (adalimumab), across all marketed dosage forms and strengths. YUFLYMA is a high-concentration, citrate-free biosimilar to Humira, approved for multiple inflammatory indications including rheumatoid arthritis (RA), psoriatic arthritis (PsA), ankylosing spondylitis (AS), ulcerative colitis (UC), plaque psoriasis (Ps), hidradenitis suppurativa (HS), and uveitis (UV) in adult patients; Crohn's disease (CD) in adults and pediatric patients 6 years of age and older; and juvenile idiopathic arthritis (JIA) in patients 2 years of age and older.[1] "This full interchangeability designation comes at a pivotal time as Celltrion continues to lead in the evolving biosimilar landscape," said Thomas Nusbickel, Chief Commercial Officer at Celltrion USA. "In alignment with recent federal initiatives aimed at lowering drug costs, we've taken timely action to enhance patient access and affordability. YUFLYMA – a high-concentration, citrate-free adalimumab biosimilar now fully interchangeable with Humira – reflects our long-standing commitment to delivering high-quality, accessible treatment options. Going forward, Celltrion will continue to put patients first by keeping drug costs affordable and remaining at the forefront of the U.S. biosimilar market, bringing competitive pricing and high-quality, accessible treatment options." The interchangeable designation builds on the Phase III interchangeability study, which demonstrated similar outcomes in terms of pharmacokinetics, efficacy, safety and immunogenicity in patients with moderately to severely active plaque psoriasis who received reference adalimumab (ADA) continuously and those who alternated between reference ADA and YUFLYMA during the dosing interval of Weeks 25-27. The result of the interchangeability study was presented at the European Academy of Dermatology & Venereology (EADV), September 2024, in the Netherlands.[2] The expanded interchangeability designation for YUFLYMA is complemented by a strategic price reduction that reflects Celltrion's ongoing commitment to improving patient access while aligning with federal efforts to lower drug prices. Effective immediately, the wholesale acquisition cost (WAC) list price of YUFLYMA has been reduced to $948 per syringe, and the updated pricing has been uploaded to the national pricing compendia. YUFLYMA was first introduced in the U.S. market in July 2023 and is currently available as a 20mg, 40mg, and 80mg solution for injection in prefilled syringes and as 40mg and 80mg in autoinjectors. Celltrion offers adalimumab-aaty in both branded and unbranded versions, with two pricing options to meet different patient needs and improve patient affordability. Notes to Editors: About YUFLYMA® (CT-P17, biosimilar adalimumab-aaty)[1] YUFLYMA is a high-concentration, low-volume and citrate-free adalimumab biosimilar to receive European Commission approval. YUFLYMA is FDA approved for the treatment of patients with rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn's disease, ulcerative colitis, plaque psoriasis, hidradenitis suppurativa and uveitis. YUFLYMA has been designated by the FDA as an interchangeable biosimilar in a prefilled syringe and autoinjector. YUFLYMA is a recombinant fully human anti–tumor necrosis factor α (anti-TNFα) monoclonal antibody. YUFLYMA is available in prefilled syringe as 20mg/0.2mL, 40mg/0.4mL and 80mg/0.8mL and autoinjector as 40mg/0.4mL and 80mg/0.8mL. Additionally, YUFLYMA features one of the longest shelf lives in its class, maintaining stability at room temperature (77 °F, 25 °C) for up to 31 days. IMPORTANT SAFETY INFORMATION[1] This important safety information also applies to YUFLYMA® (adalimumab-aaty). SERIOUS INFECTIONS Patients treated with adalimumab-aaty are at increased risk for developing serious infections that may lead to hospitalization or death. Most patients who developed these infections were taking concomitant immunosuppressants such as methotrexate or corticosteroids. Discontinue adalimumab-aaty if a patient develops a serious infection or sepsis. Reported infections include: Active tuberculosis (TB), including reactivation of latent TB. Patients with TB have frequently presented with disseminated or extrapulmonary disease. Test patients for latent TB before adalimumab-aaty use and during therapy. Initiate treatment for latent TB prior to adalimumab-aaty use. Invasive fungal infections, including histoplasmosis, coccidioidomycosis, candidiasis, aspergillosis, blastomycosis, and pneumocystosis. Patients with histoplasmosis or other invasive fungal infections may present with disseminated, rather than localized, disease. Antigen and antibody testing for histoplasmosis may be negative in some patients with active infection. Consider empiric antifungal therapy in patients at risk for invasive fungal infections who develop severe systemic illness. Bacterial, viral, and other infections due to opportunistic pathogens, including Legionella and Listeria. Carefully consider the risks and benefits of treatment with adalimumab-aaty prior to initiating therapy in patients with chronic or recurrent infection. Monitor patients closely for the development of signs and symptoms of infection during and after treatment with adalimumab-aaty, including the possible development of TB in patients who tested negative for latent TB infection prior to initiating therapy. Treatment with adalimumab-aaty should not be initiated in patients with an active infection, including localized infections. Patients over 65 years of age, patients with co-morbid conditions and/or patients taking concomitant immunosuppressants (such as corticosteroids or methotrexate), may be at greater risk of infection. Discontinue adalimumab-aaty if a patient develops a serious infection or sepsis. For a patient who develops a new infection during treatment with adalimumab-aaty, closely monitor them, perform a prompt and complete diagnostic workup appropriate for an immunocompromised patient, and initiate appropriate antimicrobial therapy. Drug interactions with biologic products: In clinical studies in patients with RA, an increased risk of serious infections has been observed with the combination of TNF blockers with anakinra or abatacept, with no added benefit; therefore, use of adalimumab-aaty with abatacept or anakinra is not recommended in patients with RA. A higher rate of serious infections has also been observed in patients with RA treated with rituximab who received subsequent treatment with a TNF blocker. There is insufficient information regarding the concomitant use of adalimumab-aaty and other biologic products for the treatment of RA, PsA, AS, CD, UC, PS, and HS. Concomitant administration of adalimumab-aaty with other biologic disease-modifying antirheumatic drugs (DMARDs) (e.g., anakinra and abatacept) or other TNF blockers is not recommended based upon the possible increased risk for infections and other potential pharmacological interactions. A higher rate of serious infections has been observed in RA patients treated with rituximab who received subsequent treatment with a TNF blocker. MALIGNANCY Lymphoma and other malignancies, some fatal, have been reported in children and adolescent patients treated with TNF blockers, including adalimumab products. Postmarketing cases of hepatosplenic T-cell lymphoma (HSTCL), a rare type of T-cell lymphoma, have been reported in patients treated with TNF blockers, including adalimumab products. These cases have had a very aggressive disease course and have been fatal. The majority of reported TNF blocker cases have occurred in patients with Crohn's disease or ulcerative colitis and the majority were in adolescent and young adult males. Almost all of these patients had received treatment with azathioprine or 6-mercaptopurine concomitantly with a TNF blocker at or prior to diagnosis. It is uncertain whether the occurrence of HSTCL is related to the use of a TNF blocker or a TNF blocker in combination with these other immunosuppressants. Consider the risks and benefits of TNF blocker treatment including adalimumab-aaty prior to initiating therapy in patients with a known malignancy other than a successfully treated non-melanoma skin cancer (NMSC), or when considering continuing a TNF blocker in patients who develop a malignancy. In controlled portions of clinical trials of some adalimumab products, more cases of malignancies have been observed compared to control-treated adult patients. NMSC was reported during clinical trials for patients treated with adalimumab products. During the controlled portions of 39 global adalimumab clinical trials in adult patients with RA, PsA, AS, CD, UC, PS, HS and UV, the rate (95% confidence interval) of NMSC was 0.8 (0.52, 1.09) per 100 patient-years among adalimumab-treated patients and 0.2 (0.10, 0.59) per 100 patient-years among control-treated patients. Examine all patients, particularly those with a medical history of prior prolonged immunosuppressant therapy or psoriasis patients with a history of psoralen + ultraviolet light A (PUVA) treatment, for the presence of NMSC prior to and during treatment with adalimumab-aaty. In clinical trials of some adalimumab products, there was an approximately threefold higher rate of lymphoma than expected in the general U.S. population. Patients with RA and other chronic inflammatory diseases, particularly those with highly active disease and/or chronic exposure to immunosuppressant therapies, may be at a higher risk (up to severalfold) than the general population for the development of lymphoma, even in the absence of TNF blockers. Postmarketing cases of acute and chronic leukemia were reported with the use of a TNF blocker in RA and other indications. Approximately half of the postmarketing cases of malignancies in children, adolescents, and young adults receiving adalimumab were lymphomas; other cases represented a variety of different malignancies and included rare malignancies usually associated with immunosuppression and malignancies that are not usually observed in children and adolescents. HYPERSENSITIVITY Anaphylaxis and angioneurotic edema have been reported following administration of adalimumab products. If an anaphylactic or other serious allergic reaction occurs, immediately discontinue administration of adalimumab-aaty and institute appropriate therapy. HEPATITIS B VIRUS REACTIVATION Use of TNF blockers, including adalimumab-aaty, may increase the risk of reactivation of hepatitis B virus (HBV) in patients who are chronic carriers. In some instances, HBV reactivation occurring in conjunction with TNF blocker therapy has been fatal. Evaluate patients at risk for HBV infection for prior evidence of HBV infection before initiating TNF blocker therapy. Exercise caution in prescribing TNF blockers for patients identified as carriers of HBV and closely monitor such patients for clinical and laboratory signs of active HBV infection throughout therapy and for several months following termination of therapy. In patients who develop HBV reactivation, stop adalimumab-aaty and initiate effective antiviral therapy with appropriate supportive treatment. The safety of resuming TNF blocker therapy after HBV reactivation is controlled is not known. Therefore, exercise caution when considering resumption of adalimumab-aaty therapy in this situation and monitor patients closely. NEUROLOGIC REACTIONS Use of TNF blocking agents, including adalimumab products, has been associated with rare cases of new onset or exacerbation of clinical symptoms and/or radiographic evidence of central nervous system demyelinating disease, including multiple sclerosis (MS) and optic neuritis, and peripheral demyelinating disease, including Guillain-Barré syndrome. Exercise caution in considering the use of adalimumab-aaty in patients with preexisting or recent-onset central or peripheral nervous system demyelinating disorders; discontinuation of adalimumab-aaty should be considered if any of these disorders develop. There is a known association between intermediate uveitis and central demyelinating disorders. HEMATOLOGIC REACTIONS Rare reports of pancytopenia including aplastic anemia have been reported with TNF blocking agents. Adverse reactions of the hematologic system, including medically significant cytopenia, have been infrequently reported with adalimumab products. Consider discontinuation of adalimumab-aaty therapy in patients with confirmed significant hematologic abnormalities. HEART FAILURE Cases of worsening congestive heart failure (CHF) and new-onset CHF have been reported with TNF blockers. Cases of worsening CHF have also been observed with adalimumab products. Exercise caution when using adalimumab-aaty in patients who have heart failure and monitor them carefully. AUTOIMMUNITY Treatment with adalimumab products may result in the formation of autoantibodies and, rarely, in the development of a lupus-like syndrome. If a patient develops symptoms suggestive of a lupus-like syndrome following treatment with adalimumab-aaty, discontinue treatment. IMMUNIZATIONS Patients on adalimumab-aaty may receive concurrent vaccinations, except for live vaccines. It is recommended that pediatric patients, if possible, be brought up to date with all immunizations in agreement with current immunization guidelines prior to initiating adalimumab-aaty therapy. No data are available on the secondary transmission of infection by live vaccines in patients receiving adalimumab products. The safety of administering live or live-attenuated vaccines in infants exposed to adalimumab in utero is unknown. Risks and benefits should be considered prior to vaccinating (live or live-attenuated) exposed infants. ADVERSE REACTIONS The most common adverse reactions in adalimumab clinical trials (>10%) were infections (e.g., upper respiratory, sinusitis), injection site reactions, headache, and rash. INDICATIONS Adalimumab-aaty is a tumor necrosis factor (TNF) blocker indicated for: Rheumatoid Arthritis (RA): reducing signs and symptoms, inducing major clinical response, inhibiting the progression of structural damage, and improving physical function in adult patients with moderately to severely active RA Juvenile Idiopathic Arthritis (JIA): reducing signs and symptoms of moderately to severely active polyarticular JIA in patients 2 years of age and older Psoriatic Arthritis (PsA): reducing signs and symptoms, inhibiting the progression of structural damage, and improving physical function in adult patients with active PsA Ankylosing Spondylitis (AS): reducing signs and symptoms in adult patients with active AS Crohn's Disease (CD): treatment of moderately to severely active Crohn's disease in adults and pediatric patients 6 years of age and older Ulcerative Colitis (UC): treatment of moderately to severely active ulcerative colitis in adults Limitations of Use: Effectiveness has not been established in patients who have lost response to or were intolerant to TNF blockers Plaque Psoriasis (Ps): treatment of adult patients with moderate to severe chronic plaque psoriasis who are candidates for systemic therapy or phototherapy, and when other systemic therapies are medically less appropriate Hidradenitis Suppurativa (HS): treatment of adult patients with moderate to severe hidradenitis suppurativa Uveitis (UV): treatment of non-infectious intermediate, posterior, and panuveitis in adult patients For Yuflyma (adalimumab-aaty): Please click for Full U.S. Prescribing Information. For adalimumab-aaty: Please see Full U.S. Prescribing Information. Globally, prescribing information varies; refer to the individual country product label for complete information. About Celltrion , Inc. Celltrion is a leading biopharmaceutical company that specializes in researching, developing, manufacturing, marketing and sales of innovative therapeutics that improve people's lives worldwide. Celltrion is a pioneer in the biosimilar space, having launched the world's first monoclonal antibody biosimilar. Our global pharmaceutical portfolio addresses a range of therapeutic areas including immunology, oncology, hematology, ophthalmology and endocrinology. Beyond biosimilar products, we are committed to advancing our pipeline with novel drugs to push the boundaries of scientific innovation and deliver quality medicines. For more information, please visit our website and stay updated with our latest news and events on our social media: LinkedIn, Instagram, X, and Facebook. About Celltrion USA Celltrion USA is Celltrion's U.S. subsidiary established in 2018. Headquartered in New Jersey, Celltrion USA is committed to expanding access to innovative biologics to improve care for U.S. patients. Celltrion's FDA-approved biosimilar products in immunology, oncology, hematology, and endocrinology include: INFLECTRA® (infliximab-dyyb), TRUXIMA® (rituximab-abbs), HERZUMA® (trastuzumab-pkrb), VEGZELMA® (bevacizumab-adcd), YUFLYMA®(adalimumab-aaty), AVTOZMA® (tocilizumab-anho), STEQEYMA® (ustekinumab-stba), STOBOCLO® (denosumab-bmwo), OSENVELT® (denosumab-bmwo) and OMLYCLO® (omalizumab-igec), as well as the novel biologic ZYMFENTRA® (infliximab-dyyb). Celltrion USA will continue to leverage Celltrion's unique heritage in biotechnology, supply chain excellence and best-in-class sales capabilities to improve access to high-quality biopharmaceuticals for U.S. patients. For more information, please visit and stay updated with our latest news and events on LinkedIn. FORWARD-LOOKING STATEMENT Certain information set forth in this press release contains statements related to our future business and financial performance and future events or developments involving Celltrion, Inc. and its subsidiaries that may constitute forward-looking statements under pertinent securities laws. This press release contains forward looking statements. These statements may be also identified by words such as "prepares", "hopes to", "upcoming", "plans to", "aims to", "to be launched", "is preparing", "once gained", "could", "with the aim of", "may", "once identified", "will", "working towards", "is due", "become available", "has potential to", "anticipate" the negative of these words or such other variations thereon or comparable terminology. In addition, our representatives may make oral forward-looking statements. Such statements are based on the current expectations and certain assumptions of Celltrion, Inc. and its subsidiaries' management, of which many are beyond its control. Forward-looking statements are provided to allow potential investors the opportunity to understand management's beliefs and opinions in respect of the future so that they may use such beliefs and opinions as one factor in evaluating an investment. These statements are not guarantees of future performance and undue reliance should not be placed on them. Such forward-looking statements necessarily involve known and unknown risks and uncertainties associated with the company's business, including the risk factors disclosed in its Annual Report and/or Quarterly Reports, which may cause actual performance and financial results in future periods to differ materially from any projections of future performance or results expressed or implied by such statements. Celltrion, Inc. and its subsidiaries undertake no obligation to update forward-looking statements if circumstances or management's estimates or opinions should change except as required by applicable securities laws. Trademarks Humira is a registered trademark of AbbVie. YUFLYMA® is a registered trademark of Celltrion, Inc., used under license. References For further information please contact:  Andria Arena [email protected] +1 516-578-0057 SOURCE Celltrion WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultDrug ApprovalPhase 3AcquisitionBreakthrough Therapy

16 May 2025

·www.businesswire.com

Neurogene Announces Evidence-Based Monitoring and Treatment Intended to Reverse Rare Hyperinflammatory Syndrome Associated with High-Dose AAV

NEW YORK--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting describing a monitoring and treatment algorithm intended to reverse the rare, severe hyperinflammatory syndrome hemophagocytic lymphohistiocytosis (HLH) that has been associated with systemic exposure to high doses of adeno-associated virus (AAV) gene therapy (>1E14 vg/kg). The 1E15 vg dose level Neurogene is moving forward in its Phase 1/2 trial of NGN-401 gene therapy for Rett syndrome translates into the E13 vg/kg range, and the Company is not aware of any case of HLH ever being reported at this dose level in AAV gene therapy. “We appreciate the opportunity that ASGCT is providing for us to share findings related to HLH and the evidence-based recommendations for how to monitor, detect and treat this rare, hyperinflammatory syndrome in the context of AAV gene therapy,” said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “As we connect with the gene therapy community to provide this information, our hope is that it spurs adoption of the early monitoring and treatment algorithm and increases propensity for sharing trial findings from which others can learn.” Early monitoring and prompt treatment have proven effective in reversing the course of HLH in the setting of higher-dose AAV gene therapy (>1E14 vg/kg)1,2. Neurogene incorporated the following monitoring and treatment protocol into its Phase 1/2 clinical trial of NGN-401 for Rett syndrome despite the use of a lower dose in the E13 vg/kg range: Implemented daily monitoring for the following in the first week post-dosing: elevated ferritin levels, fever, and falling blood counts (cytopenia), also referred to as the three Fs 3 ; over 90% of patients with HLH presented with these three initial signs seen in the HLH-2004 study 4 on which current HLH treatment guidelines are based Included HLH treatment algorithm in the trial, which includes high-dose corticosteroids as first-line treatment, and anakinra, the IL-1 receptor agonist, as second-line treatment 1 Galletta et al. J Clin Pharm Ther. 2022;47(9):1478-1481. 2 Byrne et al. Mol Ther. 2022;30(12):3503-3504. 3 Cox et al. Lancet Rheumatol. 2024;6:e51–62. 4 Bergsten et al. Blood. 2017;30(25)2728-2738. About Neurogene The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company’s novel and proprietary EXACT™ transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit www.neurogene.com. Cautionary Note Regarding Forward-Looking Statements Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the safety, tolerability and efficacy of NGN-401; the effectiveness of the monitoring and treatment protocol for HLH in Neurogene’s Phase 1/2 clinical trial of NGN-401; and the ability to reverse cases of AAV-related HLH when following this protocol. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “on track,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things, the risks and uncertainties identified under the heading "Risk Factors" included in Neurogene’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the Securities and Exchange Commission (SEC) on March 24, 2025, Neurogene’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the SEC on May 9, 2025, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

Gene TherapyClinical StudyASH

15 Apr 2025

·www.prnewswire.com

U.S. FDA grants interchangeable designation to YUFLYMA® (adalimumab-aaty), Celltrion's biosimilar to Humira® (adalimumab)

YUFLYMA ® (adalimumab-aaty) is a high-concentration (100mg/mL) and citrate-free formulation of Humira ® (adalimumab) biosimilar, that is now interchangeable [1] Interchangeable designation of YUFLYMA ® is supported by positive data from the Phase III interchangeability study in patients with moderate-to-severe plaque psoriasis INCHEON, South Korea, April 14, 2025 /PRNewswire/ -- Celltrion, Inc. today announced that the U.S. Food and Drug Administration (FDA) has designated YUFLYMA ® (adalimumab-aaty), as an interchangeable biosimilar to Humira ® (adalimumab). YUFLYMA is an FDA-approved, high-concentration (100mg/mL) and citrate-free formulation of Humira® biosimilar, approved for multiple inflammatory indications.[1] "With this new designation, YUFLYMA is further positioned to help more patients gain access to and afford the therapy they need," said Thomas Nusbickel, Chief Commercial Officer at Celltrion USA. "YUFLYMA has the same dosage form, route of administration, and dosing regimen as the reference product. The pharmacist's ability to substitute the biosimilar directly at the pharmacy without the hassle of a new prescription and without the patient having to learn a new method of administration can be a game changer in increasing patient access to adalimumab." The interchangeable designation was supported by data from the Phase III interchangeability study, which demonstrated similar outcomes in terms of pharmacokinetics, efficacy, safety and immunogenicity in patients with moderately to severely active plaque psoriasis who received reference adalimumab (ADA) continuously and those who alternated between reference ADA and YUFLYMA during the dosing interval of Week 25-27. The result of the interchangeability study was presented at the European Academy of Dermatology & Venereology (EADV) 2024.[2] FDA-approved interchangeable biosimilars may be substituted for the reference product at the pharmacy without the intervention of the prescribing health care provider, subject to state laws.[3] The high-concentration form of YUFLYMA was FDA-approved in May 2023 and is currently available as 20mg, 40 mg and 80mg solution for injection in a prefilled syringe and in an autoinjector pen. YUFLYMA was introduced into the US commercial market on July 2, 2023. YUFLYMA is available in two pricing options to help provide more affordable options for patients. Adalimumab-aaty, the unbranded version, is priced at an 85% discount to the current wholesale acquisition cost (WAC) list price of Humira (adalimumab), providing economic benefits for patients and overall healthcare system. The branded version is priced at a 5% discount to the current WAC of Humira (adalimumab). Notes to Editors: About YUFLYMA® (CT-P17, biosimilar adalimumab-aaty)[1] YUFLYMA is the world's first proposed high-concentration, low-volume and citrate-free adalimumab biosimilar to receive European Commission approval. YUFLYMA is FDA approved for the treatment of patients with rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn's disease, ulcerative colitis, plaque psoriasis, hidradenitis suppurativa and uveitis. YUFLYMA has been designated by the FDA as an interchangeable biosimilar in a prefilled syringe. YUFLYMA is a recombinant fully human anti–tumor necrosis factor α (anti-TNFα) monoclonal antibody. YUFLYMA is available in 20mg/0.2mL, 40mg/0.4mL and 80mg/0.8mL. IMPORTANT SAFETY INFORMATION[1] This important safety information also applies to YUFLYMA® (adalimumab-aaty) SERIOUS INFECTIONS Patients treated with adalimumab-aaty are at increased risk for developing serious infections that may lead to hospitalization or death. Most patients who developed these infections were taking concomitant immunosuppressants such as methotrexate or corticosteroids. Discontinue adalimumab-aaty if a patient develops a serious infection or sepsis. Reported infections include: Active tuberculosis (TB), including reactivation of latent TB. Patients with TB have frequently presented with disseminated or extrapulmonary disease. Test patients for latent TB before adalimumab-aaty use and during therapy. Initiate treatment for latent TB prior to adalimumab-aaty use. Invasive fungal infections, including histoplasmosis, coccidioidomycosis, candidiasis, aspergillosis, blastomycosis, and pneumocystosis. Patients with histoplasmosis or other invasive fungal infections may present with disseminated, rather than localized, disease. Antigen and antibody testing for histoplasmosis may be negative in some patients with active infection. Consider empiric antifungal therapy in patients at risk for invasive fungal infections who develop severe systemic illness. Bacterial, viral, and other infections due to opportunistic pathogens, including Legionella and Listeria. Carefully consider the risks and benefits of treatment with adalimumab-aaty prior to initiating therapy in patients with chronic or recurrent infection. Monitor patients closely for the development of signs and symptoms of infection during and after treatment with adalimumab-aaty, including the possible development of TB in patients who tested negative for latent TB infection prior to initiating therapy. Treatment with adalimumab-aaty should not be initiated in patients with an active infection, including localized infections. Patients over 65 years of age, patients with co-morbid conditions and/or patients taking concomitant immunosuppressants (such as corticosteroids or methotrexate), may be at greater risk of infection. Discontinue adalimumab-aaty if a patient develops a serious infection or sepsis. For a patient who develops a new infection during treatment with adalimumab-aaty, closely monitor them, perform a prompt and complete diagnostic workup appropriate for an immunocompromised patient, and initiate appropriate antimicrobial therapy. Drug interactions with biologic products: In clinical studies in patients with RA, an increased risk of serious infections has been observed with the combination of TNF blockers with anakinra or abatacept, with no added benefit; therefore, use of adalimumab-aaty with abatacept or anakinra is not recommended in patients with RA. A higher rate of serious infections has also been observed in patients with RA treated with rituximab who received subsequent treatment with a TNF blocker. There is insufficient information regarding the concomitant use of adalimumab-aaty and other biologic products for the treatment of RA, PsA, AS, CD, UC, PS, and HS. Concomitant administration of adalimumab-aaty with other biologic DMARDs (e.g., anakinra and abatacept) or other TNF blockers is not recommended based upon the possible increased risk for infections and other potential pharmacological interactions. A higher rate of serious infections has been observed in RA patients treated with rituximab who received subsequent treatment with a TNF blocker. MALIGNANCY Lymphoma and other malignancies, some fatal, have been reported in children and adolescent patients treated with TNF blockers, including adalimumab products. Postmarketing cases of hepatosplenic T-cell lymphoma (HSTCL), a rare type of T-cell lymphoma, have been reported in patients treated with TNF blockers, including adalimumab products. These cases have had a very aggressive disease course and have been fatal. The majority of reported TNF blocker cases have occurred in patients with Crohn's disease or ulcerative colitis and the majority were in adolescent and young adult males. Almost all of these patients had received treatment with azathioprine or 6-mercaptopurine concomitantly with a TNF blocker at or prior to diagnosis. It is uncertain whether the occurrence of HSTCL is related to the use of a TNF blocker or a TNF blocker in combination with these other immunosuppressants. Consider the risks and benefits of TNF blocker treatment including adalimumab-aaty prior to initiating therapy in patients with a known malignancy other than a successfully treated non-melanoma skin cancer (NMSC), or when considering continuing a TNF blocker in patients who develop a malignancy. In controlled portions of clinical trials of some adalimumab products, more cases of malignancies have been observed compared to control-treated adult patients. Non-melanoma skin cancer (NMSC) was reported during clinical trials for patients treated with adalimumab products. During the controlled portions of 39 global adalimumab clinical trials in adult patients with RA, PsA, AS, CD, UC, PS, HS and UV, the rate (95% confidence interval) of NMSC was 0.8 (0.52, 1.09) per 100 patient-years among adalimumab-treated patients and 0.2 (0.10, 0.59) per 100 patient-years among control-treated patients. Examine all patients, particularly those with a medical history of prior prolonged immunosuppressant therapy or psoriasis patients with a history of PUVA treatment, for the presence of NMSC prior to and during treatment with adalimumab-aaty. In clinical trials of some adalimumab products, there was an approximately threefold higher rate of lymphoma than expected in the general U.S. population. Patients with RA and other chronic inflammatory diseases, particularly those with highly active disease and/or chronic exposure to immunosuppressant therapies, may be at a higher risk (up to severalfold) than the general population for the development of lymphoma, even in the absence of TNF blockers. Postmarketing cases of acute and chronic leukemia were reported with the use of a TNF blocker in RA and other indications. Approximately half of the postmarketing cases of malignancies in children, adolescents, and young adults receiving adalimumab were lymphomas; other cases represented a variety of different malignancies and included rare malignancies usually associated with immunosuppression and malignancies that are not usually observed in children and adolescents. HYPERSENSITIVITY Anaphylaxis and angioneurotic edema have been reported following administration of adalimumab products. If an anaphylactic or other serious allergic reaction occurs, immediately discontinue administration of adalimumab-aaty and institute appropriate therapy. HEPATITIS B VIRUS REACTIVATION Use of TNF blockers, including adalimumab-aaty, may increase the risk of reactivation of hepatitis B virus (HBV) in patients who are chronic carriers. In some instances, HBV reactivation occurring in conjunction with TNF blocker therapy has been fatal. Evaluate patients at risk for HBV infection for prior evidence of HBV infection before initiating TNF blocker therapy. Exercise caution in prescribing TNF blockers for patients identified as carriers of HBV and closely monitor such patients for clinical and laboratory signs of active HBV infection throughout therapy and for several months following termination of therapy. In patients who develop HBV reactivation, stop adalimumab-aaty and initiate effective antiviral therapy with appropriate supportive treatment. The safety of resuming TNF blocker therapy after HBV reactivation is controlled is not known. Therefore, exercise caution when considering resumption of adalimumab-aaty therapy in this situation and monitor patients closely. NEUROLOGIC REACTIONS Use of TNF blocking agents, including adalimumab products, has been associated with rare cases of new onset or exacerbation of clinical symptoms and/or radiographic evidence of central nervous system demyelinating disease, including multiple sclerosis (MS) and optic neuritis, and peripheral demyelinating disease, including Guillain-Barré syndrome. Exercise caution in considering the use of adalimumab-aaty in patients with preexisting or recent-onset central or peripheral nervous system demyelinating disorders; discontinuation of adalimumab-aaty should be considered if any of these disorders develop. There is a known association between intermediate uveitis and central demyelinating disorders. HEMATOLOGIC REACTIONS Rare reports of pancytopenia including aplastic anemia have been reported with TNF blocking agents. Adverse reactions of the hematologic system, including medically significant cytopenia, have been infrequently reported with adalimumab products. Consider discontinuation of adalimumab-aaty therapy in patients with confirmed significant hematologic abnormalities. HEART FAILURE Cases of worsening congestive heart failure (CHF) and new-onset CHF have been reported with TNF blockers. Cases of worsening CHF have also been observed with adalimumab products. Exercise caution when using adalimumab-aaty in patients who have heart failure and monitor them carefully. AUTOIMMUNITY Treatment with adalimumab products may result in the formation of autoantibodies and, rarely, in the development of a lupus-like syndrome. If a patient develops symptoms suggestive of a lupus-like syndrome following treatment with adalimumab-aaty, discontinue treatment. IMMUNIZATIONS Patients on adalimumab-aaty may receive concurrent vaccinations, except for live vaccines. It is recommended that pediatric patients, if possible, be brought up to date with all immunizations in agreement with current immunization guidelines prior to initiating adalimumab-aaty therapy. No data are available on the secondary transmission of infection by live vaccines in patients receiving adalimumab products. The safety of administering live or live-attenuated vaccines in infants exposed to adalimumab in utero is unknown. Risks and benefits should be considered prior to vaccinating (live or live-attenuated) exposed infants. ADVERSE REACTIONS The most common adverse reactions in adalimumab clinical trials (>10%) were: infections (e.g., upper respiratory, sinusitis), injection site reactions, headache, and rash. INDICATIONS Adalimumab-aaty is a tumor necrosis factor (TNF) blocker indicated for: Rheumatoid Arthritis (RA): reducing signs and symptoms, inducing major clinical response, inhibiting the progression of structural damage, and improving physical function in adult patients with moderately to severely active RA Juvenile Idiopathic Arthritis (JIA): reducing signs and symptoms of moderately to severely active polyarticular JIA in patients 2 years of age and older Psoriatic Arthritis (PsA): reducing signs and symptoms, inhibiting the progression of structural damage, and improving physical function in adult patients with active PsA Ankylosing Spondylitis (AS): reducing signs and symptoms in adult patients with active AS Crohn's Disease (CD): treatment of moderately to severely active Crohn's disease in adults and pediatric patients 6 years of age and older Ulcerative Colitis (UC): treatment of moderately to severely active ulcerative colitis in adults Limitations of Use: Effectiveness has not been established in patients who have lost response to or were intolerant to TNF blockers Plaque Psoriasis (Ps): treatment of adult patients with moderate to severe chronic plaque psoriasis who are candidates for systemic therapy or phototherapy, and when other systemic therapies are medically less appropriate Hidradenitis Suppurativa (HS): treatment of adult patients with moderate to severe hidradenitis suppurativa Uveitis (UV): treatment of non-infectious intermediate, posterior, and panuveitis in adult patients For Yuflyma (adalimumab-aaty): Please click for Full U.S. Prescribing Information. For adalimumab-aaty: Please see Full U.S. Prescribing Information. Globally, prescribing information varies; refer to the individual country product label for complete information. About Celltrion , Inc. Celltrion is a leading biopharmaceutical company that specializes in researching, developing, manufacturing, marketing and sales of innovative therapeutics that improve people's lives worldwide. Celltrion is a pioneer in the biosimilar space, having launched the world's first monoclonal antibody biosimilar. Our global pharmaceutical portfolio addresses a range of therapeutic areas including immunology, oncology, hematology, ophthalmology and endocrinology. Beyond biosimilar products, we are committed to advancing our pipeline with novel drugs to push the boundaries of scientific innovation and deliver quality medicines. For more information, please visit our website and stay updated with our latest news and events on our social media: LinkedIn, Instagram, X, and Facebook. About Celltrion USA Celltrion USA is Celltrion's U.S. subsidiary established in 2018. Headquartered in New Jersey, Celltrion USA is committed to expanding access to innovative biologics to improve care for U.S. patients. Celltrion's FDA-approved biosimilar products in immunology, oncology, hematology, and endocrinology include: INFLECTRA® (infliximab-dyyb), TRUXIMA® (rituximab-abbs), HERZUMA® (trastuzumab-pkrb), VEGZELMA® (bevacizumab-adcd), YUFLYMA®(adalimumab-aaty), AVTOZMA® (tocilizumab-anho), STEQEYMA® (ustekinumab-stba), STOBOCLO® (denosumab-bmwo), OSENVELT® (denosumab-bmwo) and OMLYCLO® (omalizumab-igec), as well as the novel biologic ZYMFENTRA® (infliximab-dyyb). Celltrion USA will continue to leverage Celltrion's unique heritage in biotechnology, supply chain excellence and best-in-class sales capabilities to improve access to high-quality biopharmaceuticals for U.S. patients. For more information, please visit and stay updated with our latest news and events on our social media: LinkedIn. FORWARD-LOOKING STATEMENT Certain information set forth in this press release contains statements related to our future business and financial performance and future events or developments involving Celltrion, Inc. and its subsidiaries that may constitute forward-looking statements under pertinent securities laws. This press release contains forward looking statements. These statements may be also identified by words such as "prepares", "hopes to", "upcoming", "plans to", "aims to", "to be launched", "is preparing", "once gained", "could", "with the aim of", "may", "once identified", "will", "working towards", "is due", "become available", "has potential to", "anticipate" the negative of these words or such other variations thereon or comparable terminology. In addition, our representatives may make oral forward-looking statements. Such statements are based on the current expectations and certain assumptions of Celltrion, Inc. and its subsidiaries' management, of which many are beyond its control. Forward-looking statements are provided to allow potential investors the opportunity to understand management's beliefs and opinions in respect of the future so that they may use such beliefs and opinions as one factor in evaluating an investment. These statements are not guarantees of future performance and undue reliance should not be placed on them. Such forward-looking statements necessarily involve known and unknown risks and uncertainties associated with the company's business, including the risk factors disclosed in its Annual Report and/or Quarterly Reports, which may cause actual performance and financial results in future periods to differ materially from any projections of future performance or results expressed or implied by such statements. Celltrion, Inc. and its subsidiaries undertake no obligation to update forward-looking statements if circumstances or management's estimates or opinions should change except as required by applicable securities laws. Trademarks Humira is a registered trademark of AbbVie. YUFLYMA® is a registered trademark of Celltrion, Inc., used under license. References [1] YUFLYMA US prescribing information (2023) [2] Lebwohl M et al., Pharmacokinetics, Efficacy and Safety after Multiple Switches from Reference Adalimumab to Adalimumab Biosimilar (CT-P17) in comparison with the Maintenance Group (Reference Adalimumab) in Patients with Moderate-toSevere Plaque Psoriasis: Week 27 Results from the Phase III Interchangeability Study. [EADV 2024, Poster number P0931]. Available at: [3] 9 Things to Know About Biosimilars and Interchangeable Biosimilars. Available at : For further information please contact:  Andria Arena [email protected] +1 516-578-0057 SOURCE Celltrion WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalClinical ResultPhase 3

100 Deals associated with Anakinra (Amgen)

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KEGG	Wiki	ATC	Drug Bank
D02934	Anakinra (Amgen)	L04AC03	DB00026

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Approved

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Indication	Country/Location	Organization	Date
Deficiency of Interleukin-1 Receptor Antagonist	United States	Swedish Orphan Biovitrum AB	18 Dec 2020
COVID-19	European Union	Swedish Orphan Biovitrum AB	08 Mar 2002
COVID-19	Iceland	Swedish Orphan Biovitrum AB	08 Mar 2002
COVID-19	Liechtenstein	Swedish Orphan Biovitrum AB	08 Mar 2002
COVID-19	Norway	Swedish Orphan Biovitrum AB	08 Mar 2002
Cryopyrin-Associated Periodic Syndromes	European Union	Swedish Orphan Biovitrum AB	08 Mar 2002
Cryopyrin-Associated Periodic Syndromes	Iceland	Swedish Orphan Biovitrum AB	08 Mar 2002
Cryopyrin-Associated Periodic Syndromes	Liechtenstein	Swedish Orphan Biovitrum AB	08 Mar 2002
Cryopyrin-Associated Periodic Syndromes	Norway	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial cold urticaria	European Union	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial cold urticaria	Iceland	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial cold urticaria	Liechtenstein	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial cold urticaria	Norway	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial Mediterranean Fever	European Union	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial Mediterranean Fever	Iceland	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial Mediterranean Fever	Liechtenstein	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial Mediterranean Fever	Norway	Swedish Orphan Biovitrum AB	08 Mar 2002
Muckle-Wells Syndrome	European Union	Swedish Orphan Biovitrum AB	08 Mar 2002
Muckle-Wells Syndrome	Iceland	Swedish Orphan Biovitrum AB	08 Mar 2002
Muckle-Wells Syndrome	Liechtenstein	Swedish Orphan Biovitrum AB	08 Mar 2002

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Mucocutaneous Lymph Node Syndrome	Phase 3	France	Swedish Orphan Biovitrum AB	20 Oct 2023
COVID-19 respiratory infection	Phase 3	France	Swedish Orphan Biovitrum AB	27 Apr 2020
Inflammation	Phase 3	United States	Swedish Orphan Biovitrum AB	02 Apr 2020
Inflammation	Phase 3	Italy	Swedish Orphan Biovitrum AB	02 Apr 2020
Respiratory Distress Syndrome	Phase 3	United States	Swedish Orphan Biovitrum AB	02 Apr 2020
Respiratory Distress Syndrome	Phase 3	Italy	Swedish Orphan Biovitrum AB	02 Apr 2020
B-Cell Lymphoma	Phase 2	United States	Swedish Orphan Biovitrum AB	27 Dec 2021
Carney Complex	Phase 2	United States	Swedish Orphan Biovitrum AB	27 Dec 2021
Neurotoxicity Syndromes	Phase 2	United States	Swedish Orphan Biovitrum AB	27 Dec 2021
Cytokine Release Syndrome	Phase 2	United States	Swedish Orphan Biovitrum AB	01 Dec 2021

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02902731 (Pubmed \| Arthritis Res Ther)	Phase 3	Giant Cell Arteritis	-	Anakinra 100 mg	wlnquzsshf(dbnpnyyaov) = Seven serious AEs were reported in five patients, including 4 in patients receiving ANK qcmpqpxwol (qewpodcyog )	Negative	07 Jun 2025
				Placebo
NCT05498467 (CTgov)	Phase 3	Dermatitis, Allergic Contact	20	Anakinra (Anakinra)	alxguivsmn = nvglmjvunk dudqpbvmee (xkcccnufht, qyayvnjbbm - wmduesfmid) View more	-	20 May 2025
				Sodium Chloride 9mg/ml Injection (Placebo)	alxguivsmn = qngpswkiza dudqpbvmee (xkcccnufht, otkczewopx - gepqusuuee) View more
EULAR2024 Manual	Not Applicable	Still's Disease, Adult-Onset	29	Anakinra	lvgeqywxmm(wqznaslwxn) = sgrpyobkcy eyeblmkbmo (jlrgbjedjo ) View more	Positive	05 Jun 2024
EULAR2024	Not Applicable	Chondrocalcinosis IL-1β	5	Anakinra 100mg	bbvhhpftsi(ytgxwspzgp) = xcbqwrceca gobmlpqnrv (qpcyxhrgha )	Positive	05 Jun 2024
EULAR2024	Not Applicable	COVID-19	-	High dose intravenous anakinra	jgqjbvqfhk(mbbcjwnbvc) = gyiccmmlvk etyqnhmmra (mcdevmesuh ) View more	Positive	05 Jun 2024
				Anakinra (Standard of care (SoC))	qsasumminf(bjjmpfjbvy) = liivyicdwi sastgzzwvb (kllvophoew ) View more
EULAR2024	Not Applicable	COVID-19	-	High-dose intravenous anakinra + baricitinib	hggcufimmx(zpxuccckzm) = gylhlksnpk tjveqlnnpl (ctlreborgk )	Positive	05 Jun 2024
				high-dose intravenous anakinra (Control group)	hggcufimmx(zpxuccckzm) = gqpvoyxdjc tjveqlnnpl (ctlreborgk )
NCT04099901 (AFFECT-2, EHA 12024 \| EHA 22024) Manual	Phase 2	Neutropenia	88	Anakinra	wedekobtkx(herpvkuyao) = zuwmmdtfft fyjqtndijc (eqrnonbrnh ) View more	Negative	14 May 2024
				placebo	wedekobtkx(herpvkuyao) = iktqsffhhz fyjqtndijc (eqrnonbrnh ) View more
NCT04680949 (SAVE-MORE, Pubmed \| Crit Care) Manual	Phase 3	COVID-19 IL1RL1 Positive	393	Anakinra	cokewxflxq(sftbmmtgfw) = fiamxogitv sazomuhzfr (soqbhmamhj ) View more	Positive	12 Mar 2024
				Placebo	cokewxflxq(sftbmmtgfw) = gzwpubnkyr sazomuhzfr (soqbhmamhj ) View more
NCT02179853 (CTgov)	Phase 1/2	Coronary Artery Disease \| Mucocutaneous Lymph Node Syndrome	22	Anakinra (Dose Level 1 (Anakinra 2-4 mg/kg/Day))	fnrhbrjxbp = hmagayhewc trnitbwkhb (dkhjtdwfbi, fjtptdaifp - aztmthzshb) View more	-	04 Mar 2024
				Anakinra (Dose Level 1 (Anakinra 5-7 mg/kg/Day))	fnrhbrjxbp = uytuldgffy trnitbwkhb (dkhjtdwfbi, ducalyvyge - ijdbylijeg)
NCT04359784 (Tandem Meetings ASTCT and CIBMTR2024) Manual	Phase 2	B-Cell Lymphoma CD19	15	Anakinra 20Anakinra SC	hpmoojyhvt(idqfhzqdrk) = wehqjxyfya fozfpiamzb (hkrtkxbayg ) View more	Negative	01 Feb 2024
				Liso-cel alone	hpmoojyhvt(idqfhzqdrk) = mwawifdfbi fozfpiamzb (hkrtkxbayg ) View more

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