FDA Rejects Friedreich's Ataxia Drug Vatiquinone
The FDA declined PTC Therapeutics' vatiquinone for Friedreich's Ataxia, citing insufficient efficacy data and requiring more studies.
/ 2 items
The FDA declined PTC Therapeutics' vatiquinone for Friedreich's Ataxia, citing insufficient efficacy data and requiring more studies.
FDA-approved SEPHIENCE (sepiapterin), an oral drug, treats PKU in patients 1 month+. SEPHIENCE aims to be the future standard of care.