FDA Approves First Treatment for Rare Genetic Disease in Males

The FDA has approved Stealth Biotherapeutics' Forzinity injection for treating Barth syndrome, a rare and life-threatening disease affecting males. The treatment, given via subcutaneous injection, works by improving mitochondrial structure and function. The FDA approved the drug under its accelerated approval pathway, requiring additional post-market trials to confirm its effectiveness. The drug is the first treatment approved for Barth syndrome, a condition that affects approximately 230-250 males worldwide.

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Stealth Biotherapeutics' Forzinity injection for the treatment of Barth syndrome, a rare and life-threatening mitochondrial disease primarily affecting males Stealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY™ (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome^[1]. The drug, administered via subcutaneous injection, works by improving mitochondrial structure and function, addressing the core issues of the disease FDA grants first treatment approval for drug treating rare Barth syndrome^[2].

Barth syndrome is characterized by severe heart failure in infancy, leading to premature death in many cases. Survivors often face fatigue, poor stamina, and exercise intolerance, significantly impacting their daily lives and overall health. The FDA's accelerated approval pathway requires additional post-market trials to confirm the drug's effectiveness Stealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY™ (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome^[1].

Forzinity is the first treatment approved for Barth syndrome, which affects approximately 230-250 males worldwide FDA grants first treatment approval for drug treating rare Barth syndrome^[2]. The drug's approval marks a significant milestone in the treatment of ultra-rare diseases and highlights the FDA's commitment to expediting access to innovative therapies.

Stealth Biotherapeutics, a commercial-stage biotechnology company focused on mitochondrial dysfunction, has been working on Forzinity for over a decade. The company's mission is to develop novel therapies to improve the lives of patients living with diseases of mitochondrial dysfunction Stealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY™ (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome^[1]. With the FDA's approval, Stealth will continue to expand access to Forzinity, including efforts to include younger patients in clinical trials Stealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY™ (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome^[1].

The approval of Forzinity is supported by the efficacy and safety data from the TAZPOWER clinical trial. The drug has shown promising results in improving muscle strength, an intermediate clinical endpoint. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial Stealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY™ (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome^[1].