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Biomarin Submits Valoctocogene Roxaparvovec BLA to the FDA for Treatment of Hemophilia A

SAN RAFAEL, Calif., Dec. 23, 2019 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.  Subject to completion of the …

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Bayer Collaborates with Children’s Hospital of Philadelphia to Research Novel Small Molecule Therapies for Oral Treatment of Hemophilia

WHIPPANY, N.J., Dec. 5, 2019 /PRNewswire/ — Bayer announced today that it has entered into a three-year collaboration agreement with Children’s Hospital of Philadelphia (CHOP) for the discovery and development of small molecules (SMOLs) to develop a first-in-class oral non-replacement therapy (NRT) for the treatment of hemophilia A and B. …

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Takeda and Enzyre to Develop Diagnostic Device Allowing Hemophilia Patients to Determine their Coagulation Status at Home

NIJMEGEN, Netherlands, Dec. 5, 2019 /PRNewswire/ — Enzyre, which is developing breakthrough ambulant diagnostic technology for coagulation testing, today announced that it has entered into a research collaboration agreement with Takeda Pharmaceutical Company Limited (Takeda) to develop a diagnostic device that will enable hemophilia patients to determine their coagulation status …

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bluebird bio and Novo Nordisk Partner to Develop in vivo Genome Editing Candidates for Hemophilia and Other Severe Genetic Diseases

CAMBRIDGE, Mass. and BAGSVÆRD, Denmark–(BUSINESS WIRE)–bluebird bio, Inc. (Nasdaq: BLUE) and Novo Nordisk A/S (NVO) today announced that they have entered into a research collaboration to jointly develop next-generation in vivo genome editing treatments for genetic diseases, including hemophilia. During the three-year research collaboration, bluebird and Novo Nordisk will focus on identifying a development …

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FDA Approves Octapharma’s Wilate for the Treatment of Adults and Adolescents with Hemophilia A

HOBOKEN, N.J.–(BUSINESS WIRE)–Octapharma USA today announced the U.S. Food and Drug Administration (FDA) has approved WILATE® for treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and on demand treatment and control of bleeding episodes. WILATE® is a human plasma-derived, sterile, purified, double virus inactivated von Willebrand …

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Sigilon’s SIG-001 Receives Orphan Drug Designation from the FDA for Treatment of Hemophilia A

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sigilon Therapeutics today announced it has received Orphan Drug Designation for SIG-001, an investigational therapy for hemophilia A that leverages Sigilon’s Shielded Living Therapeutics™ platform to implant cells engineered to produce stable blood plasma levels of factor VIII, a crucial clotting protein. “We are very pleased to have received …

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Head-to-Head Study Demonstrated Improved PK Characteristics for Jivi Compared to Eloctate in Patients with Severe Hemophilia A

WHIPPANY, N.J., Aug. 13, 2019 /PRNewswire/ — A head-to-head study directly comparing the pharmacokinetic (PK) profiles of two extended half-life (EHL) recombinant factor VIII (FVIII) therapies demonstrated improved PK characteristics for Jivi® (antihemophilic factor [recombinant] PEGylated-aucl) compared to Eloctate® (recombinant FVIII fusion protein) in adult patients (n=17) with severe hemophilia …

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Pfizer and Sangamo’s Investigational Hemophilia A Gene Therapy Shows Sustained Increased Factor VIII Levels in Phase 1/2 Results

BRISBANE, Calif. & NEW YORK–(BUSINESS WIRE)–Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, and Pfizer, Inc. (NYSE: PFE) today announced updated results from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. The data showed that SB-525 was generally well-tolerated and demonstrated a dose-dependent …

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Sangamo and Pfizer Announce Phase 1/2 Interim Data for Investigational Hemophilia A Gene Therapy

BRISBANE, Calif. & NEW YORK–(BUSINESS WIRE)–Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, and Pfizer, Inc. (NYSE: PFE) today announced interim data from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. Data indicate that SB-525 was generally well-tolerated and demonstrated a dose-dependent increase …

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FDA Approves Novo Nordisk’s Esperoct for Treatment of Adults and Children with Hemophilia A

Bagsværd, Denmark, 19 February 2019 – Novo Nordisk today announced that the US Food and Drug Administration (FDA) has approved the Biologics License Application for ESPEROCT® for the treatment of adults and children with hemophilia A. ESPEROCT® is the brand name for turoctocog alfa pegol, N8-GP. ESPEROCT® is indicated for use in adults and …

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