Tag Archives: Hemophilia B

November, 2018

15 November
uniQure Announces Initial Topline Data from Dose-Confirmation Study of AMT-061 in Patients with Hemophilia B

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 15, 2018 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced initial clinical data in patients treated in the Company’s Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy containing …
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27 November
FDA Updates Label for Alprolix, Adding Long-Term Safety and Efficacy Data

WALTHAM, Mass.–(BUSINESS WIRE)–Bioverativ Inc. (NASDAQ: BIVV), a global biopharmaceutical company dedicated to transforming the lives of people with rare blood disorders, today announced that the U.S. Food and Drug Administration (FDA) has approved updated labeling for ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein], the leading extended half-life therapy for the …
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8 September
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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1 June
FDA Approves New Novo Nordisk Hemophilia B Treatment

PLAINSBORO, N.J., May 31, 2017 /PRNewswire/ — Novo Nordisk today announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application for REBINYN® (Coagulation Factor IX (Recombinant), GlycoPEGylated) for the treatment of adults and children with hemophilia B. Hemophilia B is a chronic and inherited bleeding …
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5 May
Three Sangamo Clinical Programs Receive Special Regulatory Designations from the FDA

RICHMOND, Calif., May 4, 2017 /PRNewswire/ — Sangamo Therapeutics, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, announced today that the Company has received notice from the U.S. Food and Drug Administration (FDA) of special regulatory designations for three of the Company’s clinical programs. Rare Pediatric Disease (RPD) designation …
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7 April
Spark’s Hemophilia B Candidate Demonstrates Positive Early-Stage Data

PHILADELPHIA, April 06, 2017 (GLOBE NEWSWIRE) — Spark Therapeutics (NASDAQ:ONCE) announced updated preliminary data today from 10 infused participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for hemophilia B. All participants have experienced consistent and sustained increases in factor IX activity following administration of the investigational therapy. …
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31 January
FDA Grants Breakthrough Therapy Designation to uniQure’s Investigational Hemophilia B Drug

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Jan. 30, 2017 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced that AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B, has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA). This designation …
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7 March
CSL’s Idelvion Approved for Treatment of Hemophilia B

KING OF PRUSSIA, Pa. — 04 March 2016 In clinical trials, IDELVION maintained factor IX activity levels above 5 percent over 14 days, resulting in a median annualized spontaneous bleeding rate (AsBR) of 0.00 IDELVION, CSL Behring’s long-acting recombinant albumin fusion protein, delivers on the company’s promise to develop and …
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14 April
World Hemophilia Day

Around the world, approximately one out of every one hundred people has a bleeding disorder, which includes hemophilia, as well as lesser-known bleeding disorders such as von Willebrand disease and factor XI deficiency. Many of these people (especially women with von Willebrand disease) have no idea about their condition. World …
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