W

ASHINGTON — 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.

Unlock this article by subscribing to STAT Plus. Try it FREE for 30 days!

SUBSCRIBE TODAY

What is it?

STAT Plus is a premium subscription that delivers daily market-moving biopharma coverage and in-depth science reporting from a team with decades of industry experience.

What's included?

  • Authoritative biopharma coverage and analysis, interviews with industry pioneers, policy analysis, and first looks at cutting edge laboratories and early stage research
  • Subscriber-only networking events and panel discussions across the country
  • Monthly subscriber-only live chats with our reporters and experts in the field
  • Discounted tickets to industry events and early-bird access to industry reports

Leave a Comment

Please enter your name.
Please enter a comment.

  • 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

Sign up for our Morning Rounds newsletter

Your daily dose of what’s new in health and medicine.

Privacy Policy