Global Blood Therapeutics (GBT) said Friday that its once-a-day pill for sickle cell disease boosted levels of oxygen-carrying hemoglobin in a little more than half of the patients treated in a Phase 3 clinical trial — results that will allow the company to file soon for accelerated approval for the treatment in the U.S.
Some outside experts, however, cautioned that it’s far from certain whether the results will translate into better health outcomes for patients themselves, even if the clinical trial achieved its main goal.
The clinical trial enrolled 274 patients with sickle cell disease and treated them with one of two doses of Global Blood’s drug, called voxelotor, or a placebo. The results: 51% of patients on the higher dose of voxoletor achieved a hemoglobin response, defined as an increase in hemoglobin of at least one gram per deciliter after 24 weeks. Just over 6% of the placebo patients had the same hemoglobin response. The difference between the two groups was statistically significant.
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