Forzinity (elamipretide), an injectable, was recently granted Accelerated approval by the US Food and Drug Administration for the treatment of Barth syndrome in patients weighing at least 30 kg. Barth syndrome is a rare hereditary mitochondrial condition that mostly affects men. It is characterised by severe heart failure in infancy, which frequently results in early death.

Stealth Biotherapeutics Inc. has been awarded approval to develop the first therapy for this illness. The FDA's accelerated approval pathway expedites the availability of medications for serious illnesses with unmet medical requirements, based on a surrogate endpoint thought to be fairly likely to predict therapeutic benefit.

Forzinity's approval was based on increased strength in the knee extensor muscle, which the government determined could predict patient benefits such as increased mobility. The medication acts by attaching to the inner mitochondrial membrane, improving mitochondrial structure and function. It is given as a daily subcutaneous injection.

As part of the rapid approval process, the manufacturer must undertake a post-approved confirmatory trial to ensure that the reported gains in muscular strength translate into genuine clinical benefit for patients. The most prevalent adverse effects recorded in clinical trials were mild-to-moderate injection site reactions, while some serious events were also observed.

The application was given a priority evaluation and an uncommon paediatric disease designation. Stealth Biotherapeutics has been granted a rare paediatric condition priority review voucher as part of its approval.