Category: Industry News

Novo Nordisk Announces Supply Shortage

Novo Nordisk today announced an important update about the Rebinyn®, Coagulation Factor IX (Recombinant), GlycoPEGylated 3000 IU vial size.  From Novo Nordisk: In May 2023, Novo Nordisk launched the Rebinyn® 3000 IU vial size, and the demand for this vial size has outpaced our expectations. This has resulted in a temporary shortage that will lead […]

Takeda Issues Voluntary Product Withdrawal

Takeda today announced that it is conducting a voluntary market withdrawal for two product lots of 650 IU VONVENDI® [von Willebrand factor] in the U.S. Takeda announced that the withdrawal is being conducted out of an abundance of caution due to misprinted product labels with the incorrect expiration date. The expiration date printed on the […]

Novo Nordisk Issues Supply Notice

Novo Nordisk provided the following update about the NovoSeven® RT (coagulation Factor VIIa, recombinant) 8 mg vial. Due to supply constraints, Novo Nordisk will be unable to provide the 8 mg vial of NovoSeven® RT from January until mid-2024. Importantly, the overall supply of NovoSeven® RT is expected to remain intact as other vial sizes are available […]

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 […]