Boston, April 3, 2024 - We have learned from Stealth BioTherapeutics that the U.S. Food and Drug Administration (FDA) has agreed to file and review the new drug application (NDA) for elamipretide for the treatment of Barth syndrome. The NDA, submitted by the drug’s sponsor Stealth BioTherapeutics, is a crucial step in BSF’s journey to advance treatments and find a cure for Barth syndrome. We are encouraged by this news!

We both celebrate this achievement and acknowledge that we have more challenges ahead than we expected.

Worth Celebrating

“Achieving this first critical milestone is a testament to the commitment of our community to never, ever give up,” says BSF Executive Director Emily Milligan. “The last six years have presented many obstacles, and through our work together we live that commitment at every turn. It is our mantra.”

Stealth BioTherapeutics has been a stalwart sponsor for this potential treatment, and we at BSF are extremely grateful that they have worked so hard to make this happen for our disease. But even they say that we would not be where we are today without the work our community and our families have done. Collectively, we held multiple listening sessions between the FDA and the Barth syndrome community; participated in the FDA meetings that Stealth BioTherapeutics has held since 2019; held nearly 200 meetings with legislators across the country; sent many letters to the FDA from BSF leaders, patients, clinicians and supporters; and submitted to the FDA a petition with nearly 20,000 signatures seeking a full, fair, and equitable review of the clinical data.

“Our community spoke, and will keep speaking,” says Kate McCurdy, Chair of the BSF Board. “There is still a long road ahead before approval, but it feels good to finally be one step closer.”

Challenges Ahead

This is not the finish line. Now that the FDA has agreed to review the NDA, we must remain committed to ensuring that it receives a full, fair, and equitable review.

We don’t yet see FDA’s full acknowledgement of how serious and life-threatening Barth syndrome is. Treatment can significantly improve the lives of those living with Barth syndrome, and today there are no treatments specific for Barth syndrome.

There is urgent unmet need for those with this devastating, ultra-rare disease that affects fewer than 150 Americans. We continue to ask the FDA to prioritize a full, fair, and equitable review of elamipretide patient data.