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FDA Grants Priority Review to Potential Sickle Cell Disease Treatment Voxelotor


The Food and Drug Administration (FDA) will review an approval application for a drug designed to target the root cause of damage from sickle cell disease (SCD).

South San Francisco, California-based Global Blood Therapeutics announced Thursday that the FDA had accepted its application seeking accelerated approval for Voxelotor, an oral drug that it developed to treat SCD. If approved, Voxelotor will be the first therapy available to patients that targets hemoglobin polymerization, the root cause of SCD damage. The agency is expected to make its decision by Feb. 26, 2020 due to a priority review, which cuts the review time from the standard 10 months to six months.

The FDA’s acceptance of our NDA for Voxelotor under Priority Review is a major milestone in the development of this investigational therapy and further illustrates the significance the Agency places on getting important and innovative treatments to individuals living with SCD as quickly as possible,” said Ted W. Love, M.D., president and chief executive officer of GBT. “We look forward to working with the FDA during this process, with the goal of potentially changing the treatment paradigm for SCD.” 

The published data from the HOPE trial showed that among 274 patients enrolled and randomized to receive the drug at 1,500mg, 900mg or placebo, 51 percent of those receiving the 1,500mg had a hemoglobin response at week 24, compared with 7 percent of those in the placebo group, according to the intent-to-treat analysis. In the 900mg group, 33 percent of patients had a hemoglobin response. In the per-protocol analysis, hemoglobin response rates were 59 percent in the 1,500mg group, 38 percent in the 900mg group and 9 percent in the placebo group. In addition, fewer patients in the high-dose group saw worsening of anemia, while seeing greater reductions in bilirubin, than those receiving placebo.

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