Speaker: Reenie McCarthy, CEO, Stealth BioTherapeutics
Reenie McCarthy: Hi, my name is Reenie McCarthy I'm the Chief Executive Officer of Stealth BioTherapeutics, a small Massachusetts-based biotech. I'm reaching out today to applaud Senators Braun and Casey and the Senate Aging Committee for hosting this important hearing to unlock hope and access to therapies for patients living with rare, progressive and serious diseases. At our company, we're developing therapies for diseases of mitochondrial dysfunction. These can be diseases of aging but all too often are diseases affecting our most vulnerable, our children. Barths syndrome is an ultra-rare genetic disease in which many children are born in acute heart failure. And 85% of children do not live to see their fifth birthday. Even for those who survive early childhood, the disease is life-limiting. Exercise, intolerance makes it challenging for kids to play with friends. Severe muscle weakness has one 10 year old saying he can't even open a bag of chips by himself and debilitating fatigue has a teenager remarking that some days it's all he can do to get up and take a shower. We've been developing our program for Barth syndrome for about 10 years and we've been asking the FDA to review our clinical data for five years. Over those past five years, while we've been talking to FDA, 10% of the Barth syndrome patient population has unfortunately died. Our regulatory journey has been challenging. We've been moved through four FDA review divisions. We've been told that FDA would refuse to file and even review our new drug application. When we thought we had a path forward based on significant improvements in parameters of heart function, we were recently told that there is no regulatory path forward that is clear at this time. We're on the verge of discontinuing our development efforts. Ultra rare diseases bring unique challenges including from a regulatory perspective and without better regulatory clarity, they are largely uninvestable. I applaud Senator Braun for introducing the Promising Pathways Act to provide better clarity on regulatory pathways for these serious rare and progressive diseases without sacrificing scientific integrity. It is crucial to understand that FDA will follow regulatory precedent consistently; that it will utilize regulatory flexibility consistency across review divisions and reviewers. We need to motivate more, not less investment in our most vulnerable patient populations. We can do better and I applaud you all for your work to bring us to that juncture. Thanks again.
