VTv Therapeutics Gets FDA Orphan Drug Designation for HPPD in Sickle Cell Disease
By Stephen Nakrosis
VTv Therapeutics said the Food and Drug Administration granted orphan drug designation to HPPD, which is being investigated as a treatment for sickle cell disease.
The FDA grants orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Sponsors of drugs receiving the designation qualify for incentives, which include tax credits, exemption for user fees, and potential market exclusivity for up to 7 years.
Preclinical studies which took place at Augusta University showed HPPD was orally active and well tolerated, the company said. The studies also showed HPPD increased fetal hemoglobin, and reduced oxidative stress and red blood cell sickling, findings which support the treatment's continued development, vTv added.
The biopharmaceutical company said it is focused on developing oral, small molecule drug candidates to help treat people living with diabetes and other chronic diseases.
Write to Stephen Nakrosis at stephen.nakrosis@wsj.com
(END) Dow Jones Newswires
October 07, 2026 17:02 ET (21:02 GMT)
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