Hemophilia Federation of America is a national nonprofit organization that assists, educates, and advocates for the bleeding disorders community.
Note: The following is edited from a press release from Sanofi. Read the full press release in its entirety here. Sanofi and Bioverativ Inc., a biopharmaceutical company focused on therapies for hemophilia and other rare blood disorders, have entered into a definitive agreement under which Sanofi will acquire all of the outstanding shares of Bioverativ for $105 […]
Note: The following is an edited version of a press release from Sigilon Therapeutics. Read the press release from Sigilon Therapeutics in it’s entirety here. Flagship Pioneering, a fully-integrated life science innovation enterprise, recently announced the launch of Sigilon Therapeutics Inc. Sigilon Therapeutics is a biopharmaceutical company that discovers and develops category-defining biocompatible encapsulated cell therapies. Sigilon Therapeutics’ discovery platform combines cell engineering and […]
Bioverativ announced on June 12, 2017 that the U.S. Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug (IND) application for BIVV001 (also known as rFVIIIFc-VWF-XTEN), a novel, investigational factor VIII therapy designed to potentially extend protection from bleeds with prophylaxis dosing of once weekly or longer for people with hemophilia A. […]
Novo Nordisk recently 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. Novo Nordisk expects to launch REBINYN® in the U.S. in the first half of 2018. For more info on REBINYN, […]
Spark Therapeutics issued a press release on updated preliminary data from 10 infused participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for hemophilia B. The data is being presented at Hemostastis & Thrombosis Research Society (HTRS) this week. The press release can be read in full here.
The following is excerpted from a press release from PRNewswire. Read the press release in it’s entirety here. Sangamo BioSciences, Inc. announced earlier this week that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SB-FIX, the company’s zinc finger nuclease (ZFN)-mediated genome editing product candidate for the treatment of hemophilia B. Sangamo […]
Note: The below is an edited version of a press release by Spark Therapeutics. The original release can be read in it’s entirety here. PHILADELPHIA, June 13, 2016 – Spark Therapeutics announced today updated results of the first cohort from the ongoing Phase 1/2 clinical trial of SPK-9001, the lead investigational candidate in its SPK-FIX program, which is being studied for the […]
Note: The below is an edited version of a press release by CSL Behring. The original release can be read in it’s entirety here. KING OF PRUSSIA, Pa., May 26, 2016 — CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has approved AFSTYLA [Antihemophilic Factor (Recombinant), Single Chain], its novel long-lasting recombinant factor […]
Note: This is an edited form of a press release from Octapharma. To read the original release in its entirety, click here. Octapharma USA announced on August 13, 2015 that the U.S. Food and Drug Administration (FDA) has approved revised product labeling for WILATE® [von Willebrand Factor/Coagulation Factor VIII Complex (Human)] to include prevention of excessive […]
Note: This is an edited version of a Emergent BioSolutions Inc. press release. To read the full release, click here. Emergent BioSolutions Inc. announced on Thursday, April 29 the U.S. Food and Drug Administration (FDA) has approved IXINITY®, an intravenous recombinant human coagulation factor IX therapeutic for the control and prevention of bleeding episodes and for perioperative management […]
In recognition of World Hemophilia Day April 17, the World Federation of Hemophilia (WFH) and CSL Behring announced today that CSL Behring has once again committed to donating bleeding disorder protein therapy international units (IUs) to the WFH Global Alliance for Progress (GAP) Program and other WFH programs, as well as making significant financial […]
The recent outbreak of Ebola virus has caused great concern among many people. We read stories about isolation, experimental therapies and many deaths. It is understandable that this raises questions for persons who are more vulnerable to infections such as persons with immunedeficencies. We have seen questions and concerns expressed by various persons and would […]
The European Commission has published approval of Octapharma’s Nuwiq ® (simoctocog alfa) for the treatment and prophylaxis of bleeding in all age groups with hemophilia A (congenital factor VIII [FVIII] deficiency). Nuwiq ® is the newest generation of FVIII replacement, produced in a human cell line cultured without additives of human or animal origin. Nuwiq […]
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