Gene therapy for severe hemophilia is approved by FDA
U.S. health regulators have approved a gene therapy for the most common form of hemophilia
WASHINGTON (AP) — U.S. officials on Thursday approved drugmaker BioMarin's gene therapy for the most common form of hemophilia, an infused treatment that can significantly reduce dangerous bleeding problems.
The Food and Drug Administration approved Roctavian for adult patients with severe cases of hemophilia A, the inherited blood-clotting disorder that can lead to bleeding after minor injuries or scrapes. It's the first gene therapy for those patients.
The IV therapy is a long-awaited alternative to current treatments, including weekly doses of a protein needed to help blood clot. Some patients take a newer, longer-acting biotech drug that replaces the protein.
BioMarin said in a statement that the FDA approval was based on a three-year study showing a 50% reduction in annual bleeding incidents among 134 patients who received the treatment. Most patients continued to respond to the treatment beyond three years, without needing regular IV infusions, the company said.
