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Hemophilia

Genentech’s Hemlibra Receives FDA Approval for Treatment of Hemophilia A with Inhibitors

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved HEMLIBRA® (emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor …

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Interim Data of Prophylactic Eloctate Shows Improvements in Long-Term Joint Health for Hemophilia A Patients

WALTHAM, Mass. & STOCKHOLM–(BUSINESS WIRE)–Bioverativ Inc. (NASDAQ: BIVV) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced the publication of interim results from a longitudinal study of joint health in patients treated prophylactically with ELOCTATE® [Antihemophilic Factor (Recombinant), Fc Fusion Protein], marketed as Elocta® in Europe and the Middle East, for treatment …

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FDA Accepts Bayer’s Biologics License Application for BAY94-9027 for Treatment of Hemophilia A

WHIPPANY, N.J., Oct. 30, 2017 /PRNewswire/ — The U.S. Food and Drug Administration (FDA) has accepted Bayer’s Biologics License Application (BLA) filing for BAY94-9027, an extended half-life site-specifically PEGylated recombinant human Factor VIII compound, for the treatment of hemophilia A in adults and adolescents 12 years of age and over. …

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Biomarin’s Gene Therapy Granted FDA Breakthrough Therapy Designation for the Treatment of Hemophilia A

SAN RAFAEL, Calif., Oct. 26, 2017 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S. Food and Drug Administration (FDA) granted valoctocogene roxaparvovec (formerly BMN 270) Breakthrough Therapy Designation. The FDA’s Breakthrough Therapy Designation program is intended to facilitate and expedite development and review of new drugs …

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Patient Death Forces Alnylam to Suspend Dosing in All Ongoing Fitusiran Studies in Hemophilia Patients

CAMBRIDGE, Mass.–(BUSINESS WIRE)– Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY), the leading RNAi therapeutics company, announced today an update on the company’s fitusiran and givosiran investigational RNAi therapeutic programs. With fitusiran, an RNAi therapeutic in development for the treatment of hemophilia A and B with or without inhibitors, Alnylam is reporting a fatal thrombotic …

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Genentech’s Emicizumab Receives Priority Review from the FDA for Treatment of Hemophilia A with Inhibitors

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) and granted Priority Review for emicizumab prophylaxis (preventative) as a once-weekly subcutaneous treatment for adults, adolescents …

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Bioverativ and Invicro Enter Imaging Collaboration to Improve Management of Joint Health in Hemophilia Patients

WALTHAM, Mass. & BOSTON–(BUSINESS WIRE)–Bioverativ Inc. (NASDAQ: BIVV), a global biotechnology company focused on the discovery, development and commercialization of innovative therapies for hemophilia and other rare blood disorders, and Invicro, LLC, a leading provider of imaging services and analysis for pharmaceutical research and development, today announced that they have formed …

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Shire Enters Bispecific Hemophilia Research and Development Deal with Novimmune

Lexington, MA – July 18, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG), the biotech leader in Rare Diseases; and Novimmune S.A., a privately-held Swiss biopharmaceutical company focused on the discovery and development of innovative, antibody-based medicines today announced they have entered into an agreement granting Shire exclusive worldwide rights to …

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Alnylam and Sanofi Genzyme Initiate Phase 3 Program of Investigational RNAi Therapeutic Fitusiran

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today the initiation of the ATLAS Phase 3 clinical program for fitusiran. The global, multicenter program is designed to evaluate the safety and efficacy of fitusiran in three separate …

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Shire Submits IND for its Gene Therapy for the Treatment of Hemophilia A

Lexington, Mass. – July 6, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG), the leading biotechnology company focused on serving people with rare diseases, today announced the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for SHP654, also designated as BAX 888, an investigational factor VIII …

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