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 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 […]
HFA Launches Clinical Trials Search Site

Hemophilia Federation of America has launched a new website to search for clinical trials for patients with bleeding disorders. Additionally, they have created a new resource for patients to learn more about the clinical trials process. “Patients and families have asked for a way to easily search for clinical trials, and Hemophilia Federation of America […]
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 […]
WFH Gene Therapy Registry: now live!

The World Federation of Hemophilia (WFH) has developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR). The launch of the WFH GTR coincides with the first approvals of gene therapy for hemophilia by both the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
From the CDC: New Hemophilia Key Findings Now Available

On December 1, the CDC recently released a new Key Findings summary of a recent scientific publication of women and girls who have hemophilia. The CDC reports, "Hemophilia primarily affects men, but women can have hemophilia, too. It was once thought that only men could have hemophilia and women could only pass on the gene that causes hemophilia […]
Children’s National Hospital Rare Disease Institute and Takeda Partner to Standardize Care for Patients With Rare Diseases
The following is an excerpt from a press release from Takeda. Read the press release in its entirety here. Children’s National Hospital and Takeda Pharmaceutical Company Limited announce the creation of the Rare Disease Clinical Activity Protocols (Rare-CAP) program, which will establish a networked system for the development, dissemination and curation of protocols to help […]
BD Announces Voluntary Recall of ChloraPrep™ 3 mL Applicator in Specific U.S. Territories and Countries
NOTE:Â The recall does not apply to any states in the United States, it only applies to the U.S. territories of Puerto Rico, Guam, U.S. Virgin Islands, Northern Mariana Islands and American Samoa. Click Below to Translate Page to Spanish. [prisna-google-website-translator] BD (Becton, Dickinson and Company), a global medical technology company, announced a voluntary recall on […]
WFH & NHF Issue Joint Statement Regarding Two Deaths Reported in Orphan Disease Gene Therapy Clinical Trial
The following is information from the World Federation of Hemophilia. A joint World Federation of Hemophilia (WFH) and National Hemophilia Foundation (NHF) statement* is issued on two deaths reported in orphan disease gene therapy clinical trial (*on behalf of the WFH Coagulation Product Safety, Supply, and Access Committee):We have become aware of two patient deaths […]