Shots: The ongoing P-III GENEr8-1 study involves assessing of valoctocogene roxaparvovec (6e13 vg/kg dose) in 134 patients with with severe hemophilia A. The patients received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up The study met 1EPs & 2EPs i.e. reduction in ABR by 84% demonstrating superiority to factor […]Read More
Shots: The FDA acceptance marks the first marketing application accepted for a gene therapy to treat hemophilia in the US. The application is based on ongoing P-III study with material from the to-be-commercialized process and updated 3yrs. P-I/II data Additionally, the FDA has accepted the PMA for an AAV5 total antibody assay intended as CDx […]Read More
Shots: The MAA submission is based on ongoing P-III study with material from the to-be-commercialized process and updated 3yrs. P-I/II data. The submission marks the first MAA submission for a gene therapy product targeting any type of hemophilia BioMarin is expected its MAA for Valoctocogene Roxaparvovec to be reviewed under expedited accelerated assessment designation in […]Read More
Shots: The P-III GENEr8-1 study results involves assessing of valoctocogene roxaparvovec (6e13 vg/kg) in 20 patients with hemophilia A The P-III GENEr8-1 study results: @23-26eks. 8 patients achieved Factor VIII levels of 40 IU/dL, mean ABR (1.5); reduction in median annualized Factor VIII usage (84%); reduction in mean FVIII usage (94%); mean & median Factor […]Read More
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