Pfizer gets US FDA nod for gene therapy in bleeding disorder
Drug is for rare genetic bleeding in hemophilia B adults (Il Sole 24 Ore Radiocor) - New York, 26 Apr - US pharmaceutical group Pfizer said the US Food and Drug Administration had approved its treatment for a rare genetic bleeding disorder for adults with hemophilia B.
The drug approved by the FDA will be marketed as BEQVEZ.
"A one-time dose of BEQVEZ has reduced bleeds post-treatment compared to standard of care with a median of zero bleeds (range 0 to 19) after up to three years of follow-up, providing sustained bleed protection and potentially avoiding years of treatment burden with prophylaxis for many patients," Pfizer said in a statement.
BEQVEZ is currently under review with the European Medicines Agency, and the treatment recently received regulatory approval in Canada, the company said.
Pfizer is looking to ramp up new innovative products following the rapid decline of its Covid business last year.
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(RADIOCOR) 26-04-24 14:20:33 (0394) 5 NNNN