FDA Denies Disc Medicine’s Rare Disease Therapy After Voucher Award
The FDA recently denied approval for bitopertin, a therapy from Disc Medicine aimed at treating a rare blood disorder known as porphyria. This rare condition significantly increases patients’ sensitivity to sunlight, leading to various health challenges.
Background of the Decision
This decision marks the first rejection of a drug processed under a new fast-track program initiated by FDA Commissioner Marty Makary. Previously, a generic antibiotic was granted approval through this expedited review in December.
Reason for Rejection
The FDA’s letter highlighted “uncertainties” regarding the relationship between a blood-based biomarker, which was used as an efficacy endpoint during clinical trials, and its actual clinical benefit for patients.
Impact on Disc Medicine
The repercussions of the FDA’s rejection were immediate. Following the announcement, shares of Disc Medicine fell by 31%, dropping to $49 during afternoon trading.
Key Takeaways
- Drug: Bitopertin
- Condition: Porphyria
- FDA Commissioner: Marty Makary
- Shares Decline: 31% drop to $49
- Approval Date of Generic Antibiotic: December (previous year)
The rejection underlines the complexities faced by companies like Disc Medicine in bringing innovative therapies to market, particularly for rare diseases. The ongoing dialogue between regulatory bodies and biotech companies is crucial for navigating these challenges.
