A US Food and Drug Administration (FDA) panel of experts has voted in support of Stealth BioTherapeutics’ elamipretide for Barth syndrome, an ultra-rare genetic disease that affects approximately 150 people in the US.
There are currently no therapies approved by the regulator for Barth syndrome, which primarily occurs in males and is characterised by cardiac abnormalities.
The disease leads to muscle weakness, exercise intolerance, fatigue, heart failure, recurrent infections and delayed growth, and is associated with a reduced life expectancy.
The Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted ten to six in favour of Stealth’s application for elamipretide, concluding that the drug is effective for this patient population.
Among the evidence supporting the committee’s decision was positive results from the TAZPOWER part two baseline-controlled extension study, the late-stage SPIBA-001 natural history control study, as well as supportive biomarker and preclinical data.
Stealth’s chief executive officer, Reenie McCarthy, said: “We are pleased that the FDA advisory committee has thoughtfully considered the elamipretide data package… and recognised that this data supports the potential of elamipretide to improve the lives of patients with this devastating disease.”
The CRDAC’s vote will now be considered by the FDA as it completes its independent review of elamipretide in this indication, with a final decision expected in January 2025.
Elamipretide has already been granted orphan drug, fast track and rare paediatric disease designations by the FDA, as well as orphan drug designation by the European Medicines Agency for Barth syndrome. The drug is also being evaluated in late-stage trials for primary mitochondrial myopathy and dry age-related macular degeneration.
Beyond elamipretide, Stealth is evaluating a topical ophthalmic formulation of its clinical-stage candidate bevemipretide for dry age-related macular degeneration, and has a pipeline of compounds under evaluation for rare neurological and cardiac disease indications.
The vote on elamipretide comes just one month after the company was awarded a grant from the Friedreich’s Ataxia (FA) Research Alliance to evaluate its mitochondria-targeted molecule SBT-589 in FA, an inherited disease that causes progressive damage to the nervous system and estimated to affect one in 50,000 individuals in the US.