UPDATE: Takeda to Voluntarily Replace Certain BAXJECT® II Reconstitution Devices
UPDATE as of 8/28/2023 Takeda, in agreement with the U.S. Food and Drug Administration (FDA), has decided to voluntarily replace BAXJECT® II reconstitution devices produced by Baxter between October 2021 and January 2022 co-packaged for use in conjunction with RECOMBINATE™ [Antihemophilic Factor (Recombinant)] and RIXUBIS® [Coagulation [Factor IX (Recombinant)]. Takeda has received reports of white […]
FDA Approves BioMarin’s Gene Therapy for Adults with Severe Hemophilia A
BioMarin Pharmaceutical Inc. today announced the U.S. Food and Drug Administration approved ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The one-time, single-dose infusion is […]
Sanofi Announces Data Showing Effective Bleed Protection in Children with Severe Hemophilia A
Pivotal data from the Phase 3 XTEND-Kids study evaluating ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] once-weekly prophylaxis, a factor VIII replacement therapy, in previously treated patients younger than 12 years of age with severe hemophilia A were presented recently in a late-breaking session at the Annual Meeting of the International Society on Thrombosis and […]
FDA Accepts Pfizer’s Review Application for Hemophilia B Gene Therapy Treatment
Pfizer Inc. announced today that the U.S. Food and Drug Administration has accepted for review Pfizer’s Biologics License Application for fidanacogene elaparvovec for the treatment of adults with hemophilia B. Fidanacogene elaparvovec is a novel, investigational gene therapy that contains a bio-engineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity variant of human coagulation […]
Pfizer Announces Positive Results from Hemophilia A and B Trial
Pfizer Inc. today announced their Phase 3 BASIS clinical trial evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and clinically meaningful effects. Marstacimab, a novel, investigational anti-tissue factor pathway inhibitor (anti-TFPI) being studied for the treatment of hemophilia A or B for people without inhibitors to Factor VIII (FVIII) or Factor IX […]
FDA Finalizes Blood Donor Eligibility Guidance
On May 11, 2023, the U.S. Food and Drug Administration released final guidance providing recommendations for evaluating blood and plasma donor eligibility using individual risk-based questions. Consistent with FDA’s January 2023 proposed guidance, the final guidance eliminates time-based donation deferrals and screening questions specific to men who have sex with men (MSM) and women who have […]
FDA issues response to Novo Nordisk’s request for concizumab approval
Recently Novo Nordisk received a Complete Response Letter from the Food and Drug Administration for concizumab. Concizumab was submitted for regulatory approval in the U.S. for the treatment of hemophilia A and B with inhibitors in August 2022. In the letter, the FDA requested additional information related to the monitoring and dosing of patients to […]
Sanofi: two published studies highlight fitusiran’s potential to address unmet needs
Two fitusiran Phase 3 studies published in The Lancet and The Lancet Haematology highlight potential to address unmet needs across all types of hemophilia Paris – April 4, 2023 – Two studies, published in The Lancet and The Lancet Haematology, evaluating the efficacy and safety of fitusiran, an investigational siRNA therapy for the prophylactic treatment of adults and adolescents with hemophilia A or B, reinforce the potential […]
BioMarin Update: FDA Extends PDUFA Target Action Date to June 30, 2023
SAN RAFAEL, Calif., March 6, 2023 — BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that it received notice this afternoon from the U.S. Food and Drug Administration (FDA) that the agency has extended review of the company’s Biologics License Application (BLA) for ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy for adults […]
FDA Approves Sanofi’s Once-Weekly ALTUVIIIO™
Source: Sanofi Press Release Sanofi announces that the U.S. Food and Drug Administration approved ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-vWF-XTEN Fusion Protein-ehtl], previously known as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy for adults and children with hemophilia A. Sanofi states that with ALTUVIIIO, they are looking to raise the bar for people with […]
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