WASHINGTON — The Food and Drug Administration will soon be alerting companies that certain gene therapies in development can qualify for less arduous review at the agency, Commissioner Scott Gottlieb said Tuesday.
Specifically, gene therapies for hemophilia, a rare disease in which blood doesn’t clot properly because it lacks certain proteins, could be evaluated based on whether therapy increases those proteins in the blood, regardless of whether the therapy actually causes the patient to bleed less.
Gottlieb said that hemophilia is the first disease the FDA will target with its new policy. An FDA spokesperson later said hemophilia was chosen because “it’s an area of a lot of development activity.”
Hi, is there any research at the moment for Canine Hemophilia type A? We have a 7 month pup indentified with Type A hemophilia and he has been in the ER for 3 days. This is the 3 rd bleeding since he was born. Had several transfusions which have not stopped the bleeding. We are desperately hoping to find a trial within Texas or close hereto to help us. We will be forever grateful if we can save this pup but also future ones