pharmaphorum September 22, 2025
Stealth BioTherapeutics has claimed FDA approval for Forzinity, the first treatment for ultra-rare disease Barth syndrome, just a few months after it was turned down by the regulator.
Forzinity (elamipretide) can be used to treat patients weighing at least 30 kg with Barth syndrome, an X-linked, inherited mitochondrial disorder that causes an enlarged and weakened heart, skeletal muscle problems and infections. It is very rare, affecting only around 150 people in the US.
The drug has had a tricky path to market, as the FDA rejected Stealth’s first marketing application in 2019 and rejected the second in May against the advice of its own advisory committee. While the Needham, Massachusetts-based company said at the time it had a path forward...
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