HFA Responds to News of FDA Action on BioMarin Gene Therapy Application
Click Below to Translate Page to Spanish. [prisna-google-website-translator]Today BioMarin announced that the U.S. Food and Drug Administration has issued a letter indicating that BioMarin’s investigational gene therapy for severe hemophilia A is not ready for approval in its present form. BioMarin says the FDA has asked for two years of follow-up data showing evidence of […]
BioMarin Receives Complete Response Letter (CRL) from FDA for Gene Therapy for Severe Hemophilia A
Click Below to Translate Page to Spanish. [prisna-google-website-translator]BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to the Company’s Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A on August 18, 2020.  The FDA issues a CRL to indicate that […]
Stimate Recall – CSL Behring & Ferring Pharmaceuticals Announces Extension of Stimate Recall to Consumer Level
Ferring Pharmaceuticals announced it was extending the recall of Stimate (desmopressin) to the consumer/user level. Stimate is manufactured by Ferring Pharmaceuticals Inc. and is distributed and sold in the United States by CSL Behring. This marks an expansion of Ferring’s initial July 2020 plans to conduct a narrower pharmacy-level recall.Under a consumer-level recall, people who […]
Stimate Recall – CSL Behring & Ferring Pharmaceuticals Response to HFA-NHF Letter
Click Below to Translate Page to Spanish. [prisna-google-website-translator]On July 21, 2020, Ferring Pharmaceuticals issued a voluntary recall of Stimate (desmopressin) nasal spray due to “superpotency” – in other words, amounts of desmopressin being higher than specified. Stimate is used to treat von Willebrand disease (VWD) and mild hemophilia A. It is licensed and distributed […]
RECALL NOTICE: Voluntary Recall of Stimate (Desmopressin Acetate)
Late today (July 21, 2020), HFA and NHF learned of a pharmacy level product recall of Stimate (desmopressin) nasal spray manufactured by Ferring Pharmaceuticals, and distributed in the U.S. by CSL Behring. Ferring has prepared a Health Hazard Safety Evaluation, warning that “there is a reasonable probability that the use of, or exposure to, certain […]
New Data From Study Reinforces Safety Profile of Genentech's Hemlibra® in People with Hemophilia A
The following is a press release from Genentech. Genentech, a member of the Roche Group announced results from the second interim analysis of the Phase IIIb STASEY study, which reinforce the safety profile of Hemlibra® (emicizumab-kxwh) characterized in the Phase III HAVEN clinical program. In the STASEY study, Hemlibra was effective with no new safety […]
Spark Therapeutics Announces Updated Data on Clinical Trial in Hemophilia A at ISTH 2020 Virtual Congress
The following is an excerpt from a press release from Spark Therapeutics. Spark Therapeutics, a member of the Roche Group and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, announced updated data from three dose cohorts of the ongoing Phase 1/2 clinical trial of investigational SPK-8011 in hemophilia […]
uniQure Announces License Agreement with CSL Behring to Commercialize Hemophilia B Gene Therapy
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 that uniQure and CSL Behring have entered into a licensing agreement providing CSL Behring with exclusive global rights to etranacogene […]
Pfizer and Sangamo Announces Updated Phase Result
The following is an excerpt from a press release from Pfizer. Read the press release in its entirety here. Pfizer Inc. and Sangamo Therapeutics Inc. 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. All five patients with […]
Sanofi and Sobi Donate up to 500 million Additional IUs of Clotting Factor
The following is an excerpt from a press release from Sanofi. Read the press release in its entirety here. Sanofi and Sobi™, together with the World Federation of Hemophilia (WFH) and WFH USA, announced an extension of their support of the WFH Humanitarian Aid Program with an additional donation of up to 500 million IUs […]
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