US FDA approves BioMarin's gene therapy for hemophilia A (updated)
29/06/2023 19:42
(Adds background in paragraph 2 & 3)
June 29 (Reuters) - The U.S. Food and Drug
Administration on Thursday approved BioMarin Pharmaceutical's
BMRN.O gene therapy for hemophilia A, the company said, giving
patients with the inherited bleeding disorder an option to
reduce regular blood infusions.
Biomarin's gene therapy, which would be the first for
hemophilia A, is a potentially one-time treatment that would
enable patients to forego or reduce infusions of factor proteins
into their bloodstream several times a week for life.
There are about 16,000 patients in the United States
with hemophilia A in which they are missing the factor VIII
clotting protein.
(Reporting by Khushi Mandowara and Bhanvi Satija in Bengaluru;
Editing by Krishna Chandra Eluri and Maju Samuel)
((Khushi.Mandowara@thomsonreuters.com;))