Disc Medicine Shares Drop 22% After FDA CRL on Bitopertin Approval
Disc Medicine’s shares plunged 22% after the FDA issued a complete response letter for bitopertin, citing insufficient evidence linking PPIX biomarker reductions to clinical benefits in EPP patients. The company expects APOLLO trial topline data in Q4 2026 and holds $791 million cash, funding operations into 2029.
1. FDA CRL for Bitopertin
The FDA issued a complete response letter for bitopertin’s NDA, acknowledging significant reductions in whole blood metal-free PPIX but finding no clear association between PPIX decreases and clinical benefits such as pain-free sun exposure. This decision delays any accelerated approval and underscores regulatory concerns over surrogate endpoint validity.
2. APOLLO Trial and Regulatory Path
Disc Medicine plans to request a Type A meeting with the FDA to clarify requirements for approval and will rely on APOLLO trial results, expected in Q4 2026, as potential evidence for traditional approval. The trial’s outcome on clinical endpoints is now critical to overcoming the FDA’s CRL objections.
3. Cash Position and Runway
As of December 31, 2025, Disc Medicine held approximately $791 million in cash, providing operational runway into 2029. This strong liquidity position supports ongoing trial activities and regulatory meetings without imminent financing needs.
4. Stock Performance and Technical Indicators
Shares fell about 22% on the CRL news but recovered 4.4% in premarket trading to $58.41, trading above its 20-day SMA and below its 100-day SMA. The RSI sits at neutral levels, MACD remains bearish, and analysts maintain a Buy consensus with an average price target of $102.