Stealth BioTherapeutics' Elamipretide Receives PDUFA Action Date Extension for Barth Syndrome Treatment
Stealth BioTherapeutics Inc., a biotechnology firm specializing in innovative therapies for mitochondrial dysfunctions, recently disclosed an extension regarding the Prescription Drug User Fee Act (PDUFA) action date for Elamipretide. The FDA has set a new review date for this promising treatment for patients with Barth Syndrome to April 29, 2025, allowing additional time for a comprehensive evaluation of supplemental information that the company recently provided.
This extension follows a favorable vote by the FDA's Cardiovascular and Renal Drugs Advisory Committee during their meeting on October 10, 2024, indicating that Elamipretide is effective for treating Barth Syndrome. On January 23, 2025, the company disclosed that the FDA needed this extended timeline due to Major Amendments submitted post the committee meeting. Notably, the FDA reported no safety concerns with Elamipretide and did not require new pre-marketing studies.
Reenie McCarthy, the Chief Executive Officer of Stealth BioTherapeutics, expressed appreciation for the FDA's thorough review process, underscoring confidence in the strength of the NDA package. The company is actively engaged with the agency to ensure that individuals living with Barth Syndrome can access this treatment promptly, should it receive approval.
Elamipretide stands out as the first of its kind, targeting mitochondrial dysfunction and heralding a hopeful advancement for Barth Syndrome therapies. Currently, no therapies have gained FDA or EMA approval for treating this ultra-rare genetic disorder. Barth Syndrome is a life-threatening condition, predominantly affecting males, characterized by severe cardiac issues, exercise intolerance, muscle weakness, and increased risk of infections. Tragically, many affected individuals face significantly reduced life expectancy, with a substantial percentage of early mortality occurring by age five.
Elamipretide boasts designations such as Orphan Drug, Fast Track, Priority Review, and Rare Pediatric Designation from the FDA, and an Orphan Drug designation from the EMA, emphasizing its critical importance in treating Barth Syndrome.
As Stealth BioTherapeutics continues to push forward with Elamipretide, they are also exploring other therapeutic avenues, including treatments for primary mitochondrial myopathy and age-related macular degeneration. The company is also assessing a second-generation candidate, Bevemipretide, through a topical formulation for dry age-related macular degeneration, aligning with their core mission to enhance the lives of those suffering from mitochondrial dysfunction-related diseases.
The extended PDUFA action date not only provides time for additional data analysis but also reflects the FDA’s commitment to ensuring safe and effective treatments reach the market. Stealth BioTherapeutics remains firmly committed to addressing the unmet medical needs of patients suffering from rare, debilitating conditions like Barth Syndrome, pending regulatory approval for Elamipretide. The increasing awareness and support for research into rare diseases highlight the vital role biotechnology plays in healthcare innovation, bringing hope to patients and families worldwide.
This extension follows a favorable vote by the FDA's Cardiovascular and Renal Drugs Advisory Committee during their meeting on October 10, 2024, indicating that Elamipretide is effective for treating Barth Syndrome. On January 23, 2025, the company disclosed that the FDA needed this extended timeline due to Major Amendments submitted post the committee meeting. Notably, the FDA reported no safety concerns with Elamipretide and did not require new pre-marketing studies.
Reenie McCarthy, the Chief Executive Officer of Stealth BioTherapeutics, expressed appreciation for the FDA's thorough review process, underscoring confidence in the strength of the NDA package. The company is actively engaged with the agency to ensure that individuals living with Barth Syndrome can access this treatment promptly, should it receive approval.
Elamipretide stands out as the first of its kind, targeting mitochondrial dysfunction and heralding a hopeful advancement for Barth Syndrome therapies. Currently, no therapies have gained FDA or EMA approval for treating this ultra-rare genetic disorder. Barth Syndrome is a life-threatening condition, predominantly affecting males, characterized by severe cardiac issues, exercise intolerance, muscle weakness, and increased risk of infections. Tragically, many affected individuals face significantly reduced life expectancy, with a substantial percentage of early mortality occurring by age five.
Elamipretide boasts designations such as Orphan Drug, Fast Track, Priority Review, and Rare Pediatric Designation from the FDA, and an Orphan Drug designation from the EMA, emphasizing its critical importance in treating Barth Syndrome.
As Stealth BioTherapeutics continues to push forward with Elamipretide, they are also exploring other therapeutic avenues, including treatments for primary mitochondrial myopathy and age-related macular degeneration. The company is also assessing a second-generation candidate, Bevemipretide, through a topical formulation for dry age-related macular degeneration, aligning with their core mission to enhance the lives of those suffering from mitochondrial dysfunction-related diseases.
The extended PDUFA action date not only provides time for additional data analysis but also reflects the FDA’s commitment to ensuring safe and effective treatments reach the market. Stealth BioTherapeutics remains firmly committed to addressing the unmet medical needs of patients suffering from rare, debilitating conditions like Barth Syndrome, pending regulatory approval for Elamipretide. The increasing awareness and support for research into rare diseases highlight the vital role biotechnology plays in healthcare innovation, bringing hope to patients and families worldwide.
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