FDA decision on Stealth's Barth syndrome drug delayed, again

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Title

FDA Further Delays Decision on Stealth BioTherapeutics’ Barth Syndrome Drug Elamipretide

Keywords

  • FDA delay
  • Stealth BioTherapeutics
  • Barth syndrome
  • Elamipretide
  • Rare disease drug approval
  • New Drug Application (NDA)
  • PDUFA date
  • Ultra-rare pediatric disease
  • Advisory committee vote
  • Patient advocacy

Key Facts

  • The FDA has once again delayed its approval decision for Stealth BioTherapeutics’ investigational drug elamipretide, which is intended to treat Barth syndrome, a life-threatening and ultra-rare genetic disease affecting fewer than 300 individuals worldwide2.
  • The Prescription Drug User Fee Act (PDUFA) date was most recently set for April 29, 2025, but the FDA missed this deadline and did not provide a new decision date24.
  • The FDA had previously postponed its decision in January 2025 to review additional analyses and requested a three-month extension after Stealth submitted new data, which was considered a major amendment2.
  • An FDA advisory committee voted 10-6 in October in favor of approving elamipretide, but the agency has previously raised concerns about the adequacy of clinical evidence, having originally issued a refusal-to-file letter in 2021 due to a lack of a single well-controlled efficacy trial2.
  • The Barth Syndrome Foundation, the primary patient advocacy group, has called for urgent FDA action, emphasizing that patients and families continue to wait for an approved therapy, as there are currently no approved treatments for Barth syndrome34.
  • Stealth BioTherapeutics has indicated that labeling discussions with the FDA are ongoing, but the company has not been given a revised action date for the decision4.
  • Elamipretide has been under development for more than a decade, with some patients receiving the drug under emergency access programs and reporting perceived benefits34.

Sources:

2. https://www.fiercebiotech.com/biotech/fda-misses-pdufa-date-stealths-ultra-rare-disease-candidate-delaying-approval-decision

3. https://www.businesswire.com/news/home/20250429986004/en/Barth-Syndrome-Foundation-Calls-for-Urgent-FDA-Action-Following-Elamipretide-Delay

4. https://www.empr.com/news/fda-decision-on-barth-syndrome-therapy-delayed-again/