uniQure Announces Clinical Hold on Hemophilia B Gene Therapy Program
The following is an excerpt from a press release from uniQure. Read the update in its entirety here. Today uniQure announced a clinical hold by the U.S. Food and Drug Administration (FDA) in its phase III HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. Per uniQure’s statement: “The clinical hold was initiated following […]
Sanofi to Resume Dosing in Fitusiran Clinical Studies in the U.S.
The following is a press release from Sanofi. Read the press release in its entirety here. Sanofi will resume fitusiran dosing in ongoing U.S. adolescent and adult clinical studies. Fitusiran is an investigational, small interference RNA therapy in development for the treatment of people with hemophilia A or B, with or without inhibitors. Sanofi’s first […]
Pfizer and Sangamo Announce Updated Results in Hemophilia A Gene Therapy
The following is an excerpt from a press release from Pfizer. Read the full press release here. Pfizer Inc. and Sangamo Therapeutics Inc., a genomic medicines company, announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec (SB-525 or PF-07055480), an investigational gene therapy for patients with severe hemophilia A. These data […]
uniQure Presents Data from HOPE-B Pivotal Gene Therapy Trial in Patients with Hemophilia B at ASH Annual Meeting
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the late-breaking presentation of initial data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five […]
Pfizer Doses First Participant In Phase 3 Study for People With Severe Hemophilia A and B with or Without Inhibitors
The following is from a press release from Pfizer. Read it in its entirety here. Pfizer Inc. announced that the first participant has been dosed in the Phase 3 BASIS study of marstacimab (PF-06741086), an anti-tissue factor pathway inhibitor (anti-TFPI) being evaluated for the treatment of people with severe hemophilia A or B, with or […]
uniQure Announces Positive Data from the HOPE-B Pivotal Trial Gene Therapy in Patients with Hemophilia B
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced positive top-line data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based […]
Octapharma announces FDA approval of updated NUWIQ® Prescribing Information to include immunogenicity data in previously untreated patients
The following is an excerpt from a statement from Octapharma. Read the statement in its entirety here. LACHEN, Switzerland- Octapharma is pleased to announce that the U.S. Food and Drug Administration (FDA) has approved an updated Prescribing Information (PI) for NUWIQ®, Octapharma’s human cell line-derived recombinant factor VIII (FVIII). NUWIQ ®Â is approved for the prevention and treatment of bleeding […]
Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia A Gene Therapy Treatment
The following is an excerpt from a statement from Pfizer. Read the statement in its entirety here. AFFINE is a global Phase 3, open-label, multicenter, single arm study that will evaluate the efficacy and safety of giroctocogene fitelparvovec in patients with moderately severe to severe hemophilia A. The primary endpoint is impact on annual bleed rate […]
CSL Behring Provides Update on Stimate Nasal Spray Availability
The following is an excerpt from an update from CSL Behring regarding the availability of Stimate Nasal Spray. Read the update in its entirety here. Ferring Pharmaceuticals Inc. initiated a voluntary, consumer-level recall of all batches of Stimate® Nasal Spray (desmopressin acetate) 1.5 mg/mL, which is distributed and sold by CSL Behring LLC. At this […]
Genentech Launches New Hemophilia Website
The following information has been provided by Genentech. “Genentech recently launched a new website GenentechHemophilia.com, dedicated to supporting the Hemophilia community. Here you can watch videos, read articles and learn how Genentech is committed to advancing all aspects of life with hemophilia A. Genentech supports the Hemophilia community through a variety of ways including a web series […]
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