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 disorder that causes severe pain upon exposure to sunlight due to a buildup of a light-sensitive molecule called protoporphyrin. Bitopertin is an oral drug designed to inhibit the glycine transporter 1, which may lower the production of protoporphyrin in red blood cells. By reducing the levels of this molecule, the treatment could potentially allow people with EPP to spend more time in the sun without pain.
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