Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
Read the original press release here. A median annualized bleeding rate (ABR) of 0.0 was reported in the overall study population during fitusiran prophylaxis (80 mg monthly) Fitusiran is a novel, investigational subcutaneously administered small interference RNA therapy, in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitor […]
Read the original press release here Investigational once-weekly efanesoctocog alfa prophylaxis met the primary efficacy endpoint providing clinically meaningful bleed protection for people with severe hemophilia A Results underscore the ability of efanesoctocog alfa to sustain normal to near-normal factor levels and the potential to transform prophylactic treatment, providing people with hemophilia A with higher […]
STAQ Pharma, Inc. has advised the Hemophilia Alliance and the National Hemophilia Foundation of a typographical printing error on a few units of lot 21114255A Desmopressin Acetate 1.5 mg/mL. This does not involve any patient safety issues. The expiration date on a few of the vials shows 2021-06-28 (June 28, 2021) when the correct expiration […]
HFA received the following announcement from CSL Behring. See full press release. FDA Accepts CSL Behring’s Biologics License Application for Etranacogene Dezaparvovec for Priority Review   If approved, etranacogene dezaparvovec would be the first gene therapy option for people living with hemophilia B This milestone underscores CSL Behring’s promise to develop and deliver a truly […]
HFA received the following press release from Sanofi. FDA grants efanesoctocog alfa Breakthrough Therapy designation for hemophilia A Efanesoctocog alfa is the first factor VIII therapy to be awarded Breakthrough Therapy designation by the FDA Designation is based on XTEND-1 Phase 3 study data demonstrating a clinically meaningful prevention of bleeds and superiority in prevention […]
HFA received this clinical program update from BioMarin. BioMarin Hemophilia A Clinical Development Program: An Update for the Hemophilia Community BioMarin is providing an update to the community regarding our ongoing gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy for hemophilia A has not been approved for use; it is in […]
HFA received the following update from Bayer. For more than 25 years, Bayer has remained committed to supporting patients around the world by keeping a constant pulse on their evolving needs as well as those of their caregivers and healthcare providers (HCPs). Recognizing the growing shift in patient use toward more recent products, such as […]
Read the original press release here: https://s28.q4cdn.com/781576035/files/doc_financials/2022/q1/Q1-2022-PFE-Earnings-Release.pdf During a recent quarterly stakeholder report, Pfizer announced that in March 2022 the US Food and Drug Administration lifted a clinical hold that had been placed on the Phase 3 AFFINE study in November 2021 following the observance of Factor VIII levels greater than 150% in some study […]
Read original press release here. LONDON, March 09, 2022 (GLOBE NEWSWIRE) — Freeline TherapeuticsHoldings plc announced that the first patient was dosed in its Phase 1/2 B-LIEVE dose-confirmation clinical trial of FLT180a for the treatment of hemophilia B, a debilitating genetic bleeding disorder caused by a deficiency in the clotting factor IX protein. FLT180a uses […]
Read the original press pelease here. Efanesoctocog alfa met primary and key secondary endpoints in pivotal study in hemophilia A, demonstrating superiority to prior factor prophylaxis treatment Once-weekly efanesoctocog alfa met primary endpoint in phase 3 study, resulting in a clinically meaningful prevention of bleeding episodes (bleed protection) In the key secondary endpoint, efanesoctocog alfa […]
An update for the hemophilia community from BioMarin, regarding the ongoing Phase 3 BioMarin hemophilia A gene therapy study, and a serious adverse event deemed, by the Independent Data Safety Monitoring Committee, unrelated to the investigational gene therapy from the phase 1/2 study. View the original Clinical Development Program Update BioMarin’s investigational gene therapy for […]
Original Source: Takeda Press Release January 31, 2022 VONVENDI [von Willebrand Factor (Recombinant)] is the First and Only Treatment Approved for Routine Prophylaxis to Reduce the Frequency of Bleeding Episodes in Adults Living with Severe Type 3 VWD Receiving On-Demand Therapy VWD is the Most Common Inherited Bleeding Disorder, Affecting up to Three Million (or […]
B-AMAZE long-term follow up data demonstrate sustained FIX expression up to 3.5 years; potential for expression levels in the normal range with relatively low vector doses B-LIEVE trial to confirm dose and immune management for planned Phase 3 pivotal trial launched one quarter ahead of latest guidance LONDON, Dec. 13, 2021 (GLOBE NEWSWIRE) — Freeline Therapeutics […]
NEW YORK & BRISBANE, Calif.–(BUSINESS WIRE)–Pfizer Inc. and Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec, an investigational gene therapy for patients with moderately severe to severe hemophilia A. The Alta study data, in patients with severe hemophilia A, are being […]
CAMBRIDGE, Mass., Dec. 13, 2021 (GLOBE NEWSWIRE) — Sigilon Therapeutics, Inc. (NASDAQ: SGTX), a biotechnology company that seeks to develop functional cures for chronic diseases through its Shielded Living TherapeuticsTM platform, today announced a strategic reprioritization to enable the Company to focus on MPS-1 and diabetes. “There have been key learnings in our Phase 1/2 […]
Press release source: uniQure and CSL Behring via Globe Newswire Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose of etranacogene dezaparvovec Etranacogene dezaparvovec also achieved secondary endpoint demonstrating statistical superiority in reduction of […]
Source:Â Sigilon press release Sigilon Therapeutics, Inc., a biotechnology company that seeks to develop functional cures for chronic diseases through its Shielded Living Therapeutics™ platform, reported that fibrosed spheres were observed during a retrieval procedure in a patient in its Phase 1/2 study of SIG-001 in severe or moderately severe hemophilia A. The SIG-001 trial had […]
The following is a news release from Pfizer. Read the full statement here (view PDF): We would like to share a recent update to our gene therapy clinical program for Hemophilia A (C3731003; AFFINE study) evaluating giroctocogene fitelparvovec, that we are developing with Sangamo. Following the observation of factor VIII (FVIII) levels greater than 150% […]
Monoject Flush Prefilled Saline Syringes by Aligned Medical Solutions: Recall – Due to Potential for Plunger to Draw Back After Air Has Been Expelled and Reintroduce Air Back into Syringe Read the full press release from the FDA Issue: Aligned Medical Solutions is recalling Cardinal Health’s Monoject Flush Prefilled Saline Syringes placed into 9,378 kits. […]
The following is a news release from Astellas Pharma Inc. Read the release in full here. Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., “Astellas”) reports that the ASPIRO clinical trial participant, who developed a serious adverse event as described on September 1, passed away on September 9. The cause of death is still pending. Astellas is gathering the relevant clinical information and […]
Important Update Regarding Availability of Stimate Source: CSL Press release Recently, CSL Behring announced that the company has chosen to end their distribution agreement with Ferring Pharmaceuticals for STIMATE® nasal spray, effectively thereby removing this medication as a valuable treatment option for patients with von Willebrand Disease (VWD) and mild hemophilia A. Background For over […]
Read the U.S. Food & Drug Administration’s announcement here. On August 4, 2021, Cardinal Health initiated a nationwide recall of approximately 267 million MonojectTM Flush Prefilled Saline Syringes (0.9% Sodium Chloride). The products have been found to reintroduce air into the syringe after the air has been expelled. This could result in injection of air […]
The following is sourced from Gilead. Read the update in its entirety “Gilead Warns of Counterfeit HIV Medication Being Distributed in the United States”. Click to view the original press release. Foster City, Calif., August 5, 2021 – Gilead Sciences has become aware of tampered and counterfeit versions of its once-daily single tablet HIV treatment regimen […]
The following is sourced from BioMarin. Read the update in its entirety “BioMarin Hemophilia A Clinical Development Program: An Update for the Hemophilia Community” View PDF | Download PDF BioMarin Hemophilia A Clinical Development Program: An Update for the Hemophilia Community BioMarin is pleased to update the community regarding our ongoing gene therapy clinical trial program […]
The following is sourced from a press release from Sigilon Therapeutics. Read the update in its entirety here. The U.S. Food and Drug Administration (FDA) has placed a clinical hold on Sigilon Therapeutics’ phase 1/2 study of SIG-001 in patients with severe or moderately severe hemophilia A. SIG-001 is a cell-based therapy, designed to carry significant […]
BioMarin is pleased to update the community regarding our ongoing gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. BioMarin Hemophilia A Clinical Development Program: An Update for the Hemophilia Community View PDF […]
Update for Hemophilia Associations BioMarin is pleased to update the community regarding our gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. BioMarin Hemophilia Update For Patient Associations View PDF | Download […]
Read the full press release from CSL Behring here. Global biotherapeutics leader CSL Behring  announced the closing of its global Commercialization and License agreement with uniQure for etranacogene dezaparvovec, a novel gene therapy for the treatment of hemophilia B. Etranacogene dezaparvovec is currently in Phase 3 clinical trials and has been shown to result in […]
Read the original press release here uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec, an investigational gene therapy for patients with hemophilia B, expired […]
Read the full release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on the Company’s hemophilia B gene therapy program after determining the Company satisfactorily addressed all issues identied by the FDA related […]
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