Centessa Pharmaceuticals plc: Centessa Pharmaceuticals Announces Positive Topline Data from Proof-of-Concept Study of SerpinPC in Severe Hemophilia A and B Patients Not on Prophylaxis finanznachrichten.de - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from finanznachrichten.de Daily Mail and Mail on Sunday newspapers.
Freeline Appoints Michael J. Parini as Chief Executive Officer and Reports Second Quarter 2021 Financial Results On track for three programs in the clinic by year end; trial. | August 17, 2021
- Full-year 2020 Total Revenues Increased 9% to $1.86 billion
- Company Dose-escalates in PHEarless, a Phase 1/2 Study with BMN 307 Gene Therapy for the Treatment of Phenylketonuria (PKU); the Third Potential Therapeutic Modality in its PKU Franchise
- During the Fourth Quarter, BioMarin Announced Benefit Maintained for Over Two Years in Children with Achondroplasia Treated with Vosoritide in Phase 3 Extension Study
- Marketing Applications for Vosoritide to Treat Children with Achondroplasia under Review in Europe and the U.S.; Committee for Medicinal Products for Human Use (CHMP) Opinion Expected in June 2021
- In January 2021, BioMarin Announced Positive Phase 3 Results with Valoctocogene Roxaparvovec in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set; Mean Annualized Bleed Rate was 0.9 in Subset Dosed More than Two Years Ago
Share this article
Share this article
SAN RAFAEL, Calif., Jan. 10, 2021 /PRNewswire/ BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants. All participants in the study received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up.
Figure 1: Box-and-Whiskers Plot of Factor VIII Activity in 4-Week Intervals, Subset Population (N=17 ) - Key: The boxes show the interquartile ranges with the lines in the boxes indicating medians. The ends of the whiskers represent the minimum and maximum values and diamonds indicate the means. - Includes only HIV-negative subjects dosed 2 or more years prior to Nov 2020 data cut date. One subject was lost t