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 […]
Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Bleeding Control in Highest Dose Cohort Through Two Years Following Hemophilia A Gene Therapy
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 […]
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