for erythropoietic protoporphyria (EPP)
FDA issued a Complete Response Letter for the bitopertin NDA in EPP on February 13, 2026. The application sought Accelerated Approval; specific deficiency details would require sponsor 8-K review.
Erythropoietic protoporphyria (EPP) is a painful condition caused by a buildup of protoporphyrin in the blood, skin, and liver, leading to severe sensitivity to sunlight. Bitopertin works by blocking glycine transporter 1 (GlyT1), which may reduce the production of protoporphyrin in red blood cells. This mechanism could potentially decrease the painful reactions to sunlight that patients with EPP experience.
The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older. The main questions it aims to answer are: * Whether bitopertin increases pain-free sunlight exposure after 6 months of treatment in participants with EPP or XLP
Source: ClinicalTrials.gov