FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity, the First Treatment for Barth Syndrome

The FDA has granted accelerated approval to Stealth BioTherapeutics’ Forzinity (elamipretide HCl) injection as the first approved treatment for Barth syndrome125.

Forzinity is indicated for adult and pediatric patients with Barth syndrome who weigh at least 30kg (66 pounds)125.

Barth syndrome is an ultra-rare, genetic mitochondrial disorder primarily affecting males; it is associated with muscle weakness, heart failure, and severely reduced life expectancy12.

The approval is based on data from the Phase II/III TAZPOWER clinical trial, where Forzinity improved knee extensor muscle strength, a surrogate outcome considered reasonably likely to predict clinical benefit12.

Forzinity is a peptide therapy that targets the inner mitochondrial membrane, binding to cardiolipin to support cellular energy production2.

Prior to Forzinity, there were no approved therapies for Barth syndrome2.

The approval follows a complex regulatory process, including a previous rejection and resubmission, with confirmation that all manufacturing and safety concerns were addressed35.

Forzinity has received Orphan Drug, Fast Track, Priority Review, and Rare Pediatric designations from the FDA, as well as Orphan Drug Designation from the European Medicines Agency2.

The most common side effects are mild injection site reactions, typically manageable with oral antihistamines or topical corticosteroids2.

Stealth BioTherapeutics received a Rare Pediatric Disease Priority Review Voucher from the FDA in connection with the approval2.

The company is working on label expansion to include children weighing under 30kg and is required to verify clinical benefit in a confirmatory trial for continued approval12.

Sources:

1. https://www.pharmaceutical-technology.com/news/stealth-bios-forzinity-finally-approved-as-first-barth-syndrome-therapy/

2. https://globalgenes.org/raredaily/fda-grants-accelerated-approval-to-stealth-for-barth-syndrom-therapy/

3. https://stealthbt.com/stealth-biotherapeutics-resubmits-new-drug-application-for-elamipretide-for-the-treatment-of-barth-syndrome/

5. https://www.biospace.com/drug-development/stealth-wins-expedited-fda-nod-for-first-ever-barth-syndrome-treatment