Updated June 21: News from Novo Nordisk, Potential Under-filling of Some Vials on the Market

medical supplies

Please read the latest update on this issue and addendum as of June 21, 2024. HFA published the original update on June 18, below: Novo Nordisk provided the following update about NovoSeven® RT, Novoeight®, and Esperoct® [antihemophilic factor (recombinant),glycopegylated-exei]. “Novo Nordisk has identified an issue on a specific production line that is manufacturing NovoSeven® RT, Novoeight®, […]

FDA Approves One-Time Gene Therapy for Adults with Hemophilia B

From Pfizer: Pfizer Inc. announced that the US Food and Drug Administration has approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing […]

HFA Project Featured in International, Peer-Reviewed Scientific Journal

DNA strand

Hemophilia Federation of America is excited to announce its work encouraging patient-centered research for females with bleeding disorders, conducted by members of its staff as well as trusted medical advisors in the bleeding disorders community, has been published in the international, peer-reviewed scientific journal Haemophilia. “Females+ with bleeding symptoms or a diagnosis of a bleeding […]

Update: Takeda to Voluntarily Replace Certain BAXJECT® II Reconstitution Devices

red blood cells

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

microscope in lab

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

blood donation

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

infusion

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 […]

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