What happened

In response to receiving Breakthrough Therapy Designation from the FDA on the company's lead compound, shares of Global Blood Therapeutics (NASDAQ:GBT), a clinical-stage biotech focused on blood-based disorders, rose 19% as of 11:15 p.m. EST on Tuesday.

So what

Global Blood announced today that the FDA has granted Breakthrough Therapy Designation to voxelotor (GBT440) as a hopeful treatment for sickle cell disease. This designation will provide Global Blood with enhanced guidance from the FDA on the drug's development and will also expedite the review process.

Here's what Ted Love, Global Blood's CEO, had to say about the news:

The FDA's decision to grant voxelotor the first Breakthrough Therapy designation for the treatment of sickle cell disease reflects a recognition of the promising efficacy and safety data we have collected to date for this investigational drug, as well as an acknowledgement of the overwhelming need for major advances over available therapies in the treatment of SCD patients. This designation is another significant milestone for GBT as we work to expedite the development of voxelotor.

Given the update, it is easy to understand why investors are cheering today.

Team of doctors looking over data

Image source: Getty Images.

Now what

A few weeks ago, Global Blood's shares sold off hard after investors learned that two patients who were using voxelotor through a compassionate use program had died. Predictably, that news caused a few traders to become skeptical about the drug's safety. Today's regulatory update should help to at least partially alleviate those concerns.

The next big announcement for Global Blood is expected to be made within the first half of this year. That's when the company plans on announcing data from the first part of its phase 3 HOPE study. If all goes well with that data readout, then the drug could be in regulators' hands soon after. 

Global Blood's management team will be giving a presentation at the J.P. Morgan Healthcare Conference at 7:30 a.m. PT on Wednesday. Bulls and bears alike will want to listen in for clues as to what the future may hold for this business.

Brian Feroldi has no position in any of the stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy.