Drug regulator OKs new therapy for some hemophilia patients
Despite FDA approval, Hem lib ra continues to be tested
TRENTON, N.J.—U.S. regulators have approved the first new treatment inn early two decades to prevent internal bleeding in certain patients with hemophilia.
The U.S. Food and Drug Administration has approved Hemlibra, a weekly self-injection for hemophilia A patients who have developed resistance to standard medicines for preventing bleeding episodes.
The list price will be about US$482,000 for the first year and slightly less after that, said California-based Genentech, which developed the drug.
Gen en tech, the biotech subsidiary of Swiss drug maker Roche, says that’ s half the cost of the only other preventive option for patients with this problem. That treatment requires a two-hour IV drip several times a week.
About 20,000 Americans — mostly males — have hemophilia, an inherited, potentially lifethreatening disorder. Their blood doesn’t clot properly because of a faulty gene. In severe cases, repeated bleeding in the joints leads to problems walking and intense pain. About 80 per cent of hemophilia patients have hemophilia A and about one-third of those develop resistance.
The FDA gave exp edited approval to Hemlibra, also known a semi cizumab, for adults and children who have that resistance. Genentech continues to test Hemlibra, with the aim of getting it approved for all hemophilia A patients.
In a company-funded study, participants who got Hemlibra had a 79 per cent reduction in the number of bleeding episodes compared to how they had previously fared on the older treatment. The FDA noted the patients getting Hemlibra reported less pain, joint swelling and difficulty walking.
Hemlibra carries the FDA’s strongest level of warning because it can cause severe blood clots. Common side effects include headache, joint pain and injection site reactions.
Greg Hogan’s son, Patrick, was diagnosed with hemophilia after birth. At nine months, Patrick developed such severe resistance to his medicine that he had to get massive quantities through an IV every morning. Still, the family had to severely limit his activity.
Patrick started receiving Hemlibra nine months ago as part of the testing. His father said he improved immediately, feeling stronger and behaving “more like a rambunctious child.”