FDA Accepts CSL Behring’s Biologics License Application for Etranacogene Dezaparvovec for Priority Review
Global biotherapeutics leader CSL Behring, a business of CSL, announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA), for priority review, for etranacogene dezaparvovec (also known as CSL222), an investigational gene therapy for the treatment of adults with hemophilia B. Etranacogene dezaparvovec was specifically designed to make near-normal […]
FDA Grants Breakthrough Therapy Designation for Hemophilia A
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 […]
BioMarin Issues Clinical Program Update: Hemophilia A
Bayer Discontinues Kogenate® FS in the United States
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 […]
Pfizer Reports First Quarter 2022 Update of Gene Therapy Trial
Read the original press release. During a recent quarterly stakeholder report, Pfizer announced that in March 2022 the U.S. 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 participants. Pfizer […]
Freeline Announces First Patient Dosed in Hemophilia B Trial
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 […]
Sanofi and SOBI Announce Results of XTEND-1 Phase 3 Study
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 […]
BioMarin provides update to ongoing Phase 3 hemophilia A gene therapy study
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 […]
FDA Approves Prophylactic Treatment with VONVENDI® [von Willebrand Factor (Recombinant)] for Adult Patients Living with Severe Type 3 von Willebrand Disease (VWD)
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 […]
Freeline Presents Long-Term Follow-Up Data from Phase 1/2 B-AMAZE Trial in Hemophilia B at the 2021 ASH Annual Meeting and Announces Early Initiation of Phase 1/2 B-LIEVE Dose-Confirmation Trial
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 […]
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