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Disc Medicine Announces Receipt of FDA Commissioner's National Priority Voucher (CNPV) for Bitopertin in Erythropoietic Protoporphyria (EPP)

IRON
  • Disc Medicine received a Commissioner's National Priority Voucher (CNPV) from the FDA for bitopertin in erythropoietic protoporphyria (EPP) & X-linked protoporphyria (XLP).
  • The CNPV program is designed to reduce drug application review times to 1-2 months for treatments aligned with national health priorities.
  • Disc submitted a New Drug Application (NDA) in September 2025 for patients aged 12 years & older under the FDA's accelerated approval pathway.
  • Bitopertin holds Orphan Drug Designation & Rare Pediatric Disease Designation from the FDA.
  • EPP is a rare disease caused by heme biosynthesis mutations leading to toxic protoporphyrin IX accumulation, causing severe sunlight sensitivity & potential liver failure in 20-30% of patients.
  • Bitopertin is an investigational oral GlyT1 inhibitor intended to modulate heme biosynthesis & may be the first disease-modifying therapy for erythropoietic porphyrias.
  • Bitopertin has been studied in multiple clinical trials including Phase 2 BEACON, AURORA, HELIOS, & Phase 3 APOLLO trials.
  • Disc obtained global rights to bitopertin via a license agreement with Roche in May 2021.
  • The drug remains investigational & is not approved for use in any jurisdiction worldwide.