Global Blood Therapeutics (GBT) is taking an unprecedented regulatory gamble to win approval for a new drug to treat sickle cell disease. Are investors underestimating the risk that this effort blows up?

It looks that way. The South San Francisco-based biotech disclosed mixed results from a Phase 3 clinical trial in late June. Since then, Global Blood executives have been on the road with investors, talking up an FDA submission strategy for its drug, called voxelotor, that has never been tried before in sickle cell disease.

Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free!


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.

Sign up for our Daily Recap newsletter

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy