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Stealth BioTherapeutics Announces FDA Approval of FORZINITY™ the First Therapy for rare, life-limiting pediatric mitochondrial disease
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An exciting landmark was reached with the accelerated approval of FORZINITY™ (elamipretide HCl) by the U.S. Food and Drug Administration (FDA), a significant achievement for Stealth BioTherapeutics Inc.

This approval marks a pivotal moment for the Barth syndrome community, as FORZINITY becomes the first-ever FDA-approved treatment for this rare, life-limiting pediatric mitochondrial disease.

Affecting approximately 150 individuals in the United States, Barth syndrome is characterized by severe symptoms including debilitating muscle weakness, exercise intolerance, and chronic fatigue, with many patients not surviving past the age of five. This new therapy is also the first FDA-approved therapeutic to specifically target mitochondrial dysfunction.

The approval is based on compelling efficacy and safety data from the TAZPOWER clinical trial, which demonstrated that FORZINITY significantly improved muscle strength, specifically knee extensor strength. While the most common side effects were injection site reactions, the drug's clinical benefit was clear to both patients and physicians. This accelerated approval is contingent upon the verification of clinical benefits through a confirmatory trial, a standard process for this type of regulatory decision. The FDA's decision follows a period of close collaboration between the agency and Stealth BioTherapeutics, ultimately reflecting a shared commitment to providing timely access to a community in desperate need of a treatment option.

The positive impact of this approval extends beyond just the drug itself. Leaders within the Barth syndrome community, including Stealth's CEO Reenie McCarthy and Board Chair of the Barth Syndrome Foundation Kate McCurdy, celebrated the approval as a significant victory and a beacon of hope for other ultra-rare diseases. However, they also highlighted the ongoing challenge that the current approval is limited to patients weighing at least 30 kilograms. This restriction leaves out many of the youngest and most vulnerable patients who are too small to meet the weight requirement. In response, Stealth has committed to working closely with the FDA to collect additional data to support a label expansion, while continuing to provide access to these children through their existing expanded access program. This pledge ensures that the company remains dedicated to developing therapies for all patients with this devastating disease.

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