Tags : Hemophilia A


Pfizer and Sangamo’s Giroctocogene Fitelparvovec (SB-525) Demonstrate Positive Effect in

Shots: Pfizer and Sangamo report follow-up data from the P-I/II Alta study assessing giroctocogene fitelparvovec (SB-525, or PF-07055480) in 11 patients aged 18-47yrs. with severe hemophilia A 5 patients with severe hemophilia A who received the 3e13 vg/kg dose showed sustained factor VIII (FVIII) activity levels, with a median of 64.2% via chromogenic assay as […]Read More


BioMarin Reports Submission of MAA to EMA for Valoctocogene Roxaparvovec

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


Bluebird Bio Signs a Three-Year Research Collaboration with Novo Nordisk

Shots: Bluebird Bio to utilize its mRNA-based megaTAL technology providing a specific and efficient way to silence, edit or insert genetic components combining Novo Nordisk’s deep expertise in hemophilia research and therapeutics  The focus of the collaboration is to identify gene therapy candidates for people with severe genetic diseases with initial interest of correcting FVIII-clotting […]Read More


Novo Nordisk’s Esperoct (turoctocog alfa pegol, N8-GP) Receives European Commission

Shots: The approval is based on results from pre-registration clinical programme assessing Esperoct in 270 patients with severe hemophilia A with 5+years of clinical exposure. The authorization covers all 28 European Union member states The marketing authorization follows EMA’s CHMP positive opinion granted on Apr 26, 2019 with its expected launch in European countries in […]Read More


BrightInsight Launches a CE-Marked Dosing Calculator for Emicizumab to Treat

Shots: BrightInsight’s CE-marked dosing calculator enables health care professionals to determine the correct dosage & number of vials based on recommended dosage for the treatment of Hemophilia A with its development & distribution sponsored by Roche The calculator helps in determining the correct loading and maintenance dose of emicizumab based on BrightInsight’s Internet of Things […]Read More


Novo Nordisk’s Esperoct (turoctocog alfa pegol, N8-GP) Receives CHMP’s Positive

Shots: The positive opinion is based on largest pre-registration clinical programme assessing Esperoct (FD, IV, q4days) in 270 patients previously treated with severe hemophilia A and more than 5 years of clinical exposure   The study resulted in effective routine prophylaxis in people with severe hemophilia A, efficacious in treatment and control of bleeding episodes […]Read More