Global Blood just might pull this off

Last year, when Global Blood Therapeutics said it would submit a sickle cell disease drug for FDA approval based on secondary evidence, people were more than a little skeptical. Then, in June, when the company did just that, experts warned there was no way to be sure its drug would actually improve patient outcomes.

Then, yesterday, Global Blood said that not only had the FDA accepted that application, but the agency was “not currently planning” to convene a panel of outside experts to weigh in on the drug.

That would suggest the FDA is comfortable with the idea that increasing hemoglobin, which Global Blood’s drug can do, will translate to a long-term benefit for people with sickle cell, something Global Blood’s clinical trials did not demonstrate.

The company’s share price rose about 15% on the news, as investors inferred that Global Blood’s drug, voxelotor, is now that much likelier to win approval. We'll find out in about six months.