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Biotech Innovations
February 11, 2020

Investigational Hemophilia A Gene Therapy Shows Sustained Benefit

JAMA. 2020;323(6):499. doi:10.1001/jama.2020.0470

An experimental gene therapy for hemophilia A remained effective up to 3 years after a single infusion, researchers recently reported in the New England Journal of Medicine. BioMarin Pharmaceutical’s valoctocogene roxaparvovec encodes factor VIII, the blood-clotting protein that’s missing or low in people with hemophilia A, the most common form of the disease.

“This latest study confirms both safety and the long-term beneficial impact of the treatment,” John Pasi, MD, PhD, director of the Haemophilia Centre at the Royal London Hospital and the trial’s principal investigator, told JAMA.

The study looked at 2- and 3-year data from 15 men with severe disease who received a single valoctocogene roxaparvovec infusion. All 15 patients continued not taking standard prophylactic factor VIII therapy during the follow-up period. The 13 participants who received the highest treatment doses had substantially fewer bleeding events, and all but 1 also had fully resolved bleeding in their major joints, which over time causes permanent damage. No new safety events occurred during follow-up.