Global Blood Therapeutics Gets FDA Approval for Supplemental NDA

By Chris Wack

Global Blood Therapeutics Inc. said the U.S. Food and Drug Administration has accepted for filing and review the company's supplemental New Drug Application seeking accelerated approval for Oxbryta voxelotor for the treatment of sickle cell disease in children ages 4 to 11 years.

The company said the FDA also accepted its New Drug Application seeking approval for a new age-appropriate dispersible tablet dosage form of Oxbryta suitable for pediatric patients.

Oxbryta, a first-in-class therapy that directly targets hemoglobin polymerization, the root cause of red blood cell sickling in SCD, is currently approved by the FDA in a tablet dosage form to treat SCD in patients age 12 years and older.

The FDA granted Priority Review for both the NDA and sNDA, providing a six-month review, and assigned a Prescription Drug User Fee Act target action date for both applications of Dec. 25.

The company said the NDA seeks approval for 300 mg dispersible tablets. The dispersible tablet formulation includes grape flavoring, is intended to be dispersed in room-temperature drinking water or other clear drinks for ease of swallowing, and allows for weight-based dosing in pediatric patients ages 4 to 11 years with SCD.

Global Blood shares were up 3%, to $30.98, in premarket trading.

Write to Chris Wack at [e-mail]

(END) Dow Jones Newswires

September 07, 2021 08:30 ET (12:30 GMT)

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