FDA approves first mitochondrial disease drug
The FDA last month approved elamipretide as the first therapy targeting a mitochondrial defect, for the ultra‑rare Barth syndrome that can be fatal in infancy. The decision follows earlier rejections and comes amid an active FDA pipeline — including a recent rejection of a PDCD therapy and ongoing review of MT1621 for TK2 deficiency — as researchers and biotechs expand clinical programs for mitochondria‑related conditions.
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📌 Key Facts
- FDA approved elamipretide for Barth syndrome, marking the first therapy directed at a mitochondrial flaw.
- Barth syndrome affects roughly 150 people in the United States; the approval followed prior FDA rejections and extended back‑and‑forth.
- FDA recently rejected a PDCD therapy and is currently considering MT1621 for TK2 deficiency (~250 patients worldwide).