Stealth BioTherapeutics Provides Commercial Launch Update and Pipeline Progress Across Mitochondrial Disease Portfolio

Stealth BioTherapeutics Inc, a commercial-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today provided an update on the early commercial demand for FORZINITY and highlighted continued progress across its clinical and research pipeline.

Reenie McCarthy, Chief Executive Officer of Stealth BioTherapeutics, said we are encouraged that the strong early momentum of the FORZINITY launch is facilitating prompt access for patients living with Barth syndrome. They continue to advance our pipeline in rare and age-related diseases of mitochondrial dysfunction, including dry age-related macular degeneration, positioning Stealth at the forefront of the emerging therapeutic modality of mitochondrial medicine.

According to Towards Healthcare, the biotherapeutics market is projected to experience significant growth, with estimates suggesting the market size will increase from USD 533.51 billion in 2026 to approximately USD 1241.63 billion by 2035, representing a compound annual growth rate (CAGR) of 9.84% from 2026 to 2035. Growth is driven by a robust growth trajectory for the biotherapeutics and biopharmaceutical markets, driven by the increasing prevalence of chronic diseases and the need for innovative therapies. The market's growth is expected to continue as advancements in biotechnology and drug development continue to drive demand for targeted therapies. 

About FORZINITY (elamipretide) injection 

Forzinity is indicated for improving muscle strength in adult and pediatric patients with Barth syndrome who weigh at least 30 kg. This condition is a rare genetic disorder that affects mitochondrial function, leading to muscle weakness and other complications.  

It is supplied as a sterile, clear, colorless to yellow aqueous solution in single-patient-use vials, with a concentration of 280 mg/3.5 mL (80 mg/mL). It represents the first FDA-approved therapy specifically for Barth syndrome, offering a targeted approach to improve muscle strength by addressing the underlying mitochondrial dysfunction. 

Approval is under accelerated approval based on improvement in knee extensor muscle strength, an intermediate clinical endpoint, with continued approval contingent on confirmatory trials demonstrating clinical benefit.  

A recent report by Towards Healthcare highlights that the biotherapeutics market is witnessing growth due to the increasing burden of chronic diseases and rare diseases, which is prompting the need for more effective biologics. There is a growing need for biologics that can target specific diseases, leading to a surge in the development of new therapies. With technological advancements and innovations in biotechnology and gene therapy, the treatment of various diseases is driving the demand for biotherapeutics.