In the News
Global Blood, FDA agree on fast-filing plan for new sickle cell disease drug
Source: STAT
Global Blood Therapeutics (GBT) said Monday that it had reached an agreement with the Food and Drug Administration for an accelerated approval submission of its experimental drug to treat sickle cell disease.
It is a major win for the South San Francisco-based biotech — and a sign of new flexibility at the FDA, which will allow the company to try to secure faster approval for its drug in a way never used before in sickle cell disease.
The submission plan agreed to by the FDA will encompass data from a completed clinical trial showing the drug, voxelotor, safely increases hemoglobin, the oxygen-carrying molecule in red blood cells. By raising hemoglobin, Global Blood believes voxelotor is reasonably likely to reduce the frequency of strokes in sickle cell disease patients.
But under the accelerated approval pathway, the company won’t need to conduct a clinical trial proving voxelotor reduces strokes until after the drug is approved.
Traditionally, the FDA has only approved a drug for sickle cell disease based on scientific proof demonstrating a reduction in the frequency of sickle “crises.” Also known by the shorthand “VOCs,” these are the searingly painful and potentially life-threatening exacerbations of the disease that occur when misshapen red blood cells clump inside blood vessels.