Stealth BioTherapeutics Corp, announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Adminstration (FDA) for elamipretide, the company's lead product candidate, for the treatment of Barth syndrome, an ultra-rare genetic condition with no FDA- or EMA-approved therapies.
"Children and young adults affected by Barth syndrome are suffering from life limiting, progressive cardiomyopathy, exercise intolerance, and debilitating fatigue for which there are no approved treatment options," said Chief Executive Officer Reenie McCarthy. "We initiated our Barth syndrome development efforts at the request of the Barth syndrome community. We respect the patient community's perspective regarding its tolerance for some uncertainty of benefit in considering therapies for this ultra rare disease, and with our NDA submission, have answered its petition that we submit our application. We know that the FDA has similarly heard the voice of these patients, and we look forward to continued dialogue with the FDA as it evaluates our submission for filing and review."
The NDA submission is based on results from the SPIBA-001 Phase 3 Retrospective Natural History Control Trial, which compared data from the open-label portion of the TAZPOWER Phase 2/3 clinical trial to matched natural history controls. SPIBA-001 met its primary and most secondary endpoints, demonstrating elamipretide-mediated improvements in assessments of exercise tolerance, strength and cardiac function that are unexpected in the natural course of this progressively debilitating disease. In addition, improvements were observed during the TAZPOWER Phase 2/3 clinical trial and open label extension in several surrogate and intermediate clinical endpoints that may be reasonably likely to predict clinical benefit for patients suffering from this serious disease, potentially supporting an accelerated approval pathway. Although the FDA has recommended that additional controlled data be generated to support NDA review, neither the FDA nor the Company has identified a feasible trial design due to the ultra-rare nature of this disease. In light of FDA's view that the existing clinical data are insufficient to demonstrate substantial evidence of effectiveness and would not support NDA review, there is no assurance that the FDA will file the NDA. Stealth believes, however, that the data could support an NDA review, and has accordingly submitted the NDA as requested by the Barth syndrome patient community.
Elamipretide was previously granted rare pediatric designation, fast track designation, and orphan drug designation by the FDA, and orphan drug designation by the EMA, for the treatment of Barth Syndrome.
About Barth Syndrome
Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities often leading to heart failure and reduced life expectancy, recurrent infections, muscle weakness and delayed growth. Barth syndrome occurs almost exclusively in males and is estimated to affect one in 200,000 to 400,000 individuals worldwide. There are currently no FDA- or EMA-approved therapies for patients with Barth syndrome.
https://en.wikipedia.org/wiki/Elamipretide
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