Dear Barth Families and Friends,
As you may have seen already in Stealth BioTherapeutics’ press release this morning, the date by which FDA will let Stealth know whether they will approve elamipretide (the “PDUFA date”) has been delayed by three months. We had expected to know FDA’s decision by January 29 but now have learned that the new date is April 29, 2025.
The date has been moved by the FDA because the Agency asked Stealth for a large number of new analyses after the positive Ad Comm meeting in October – enough to qualify as a “major amendment” to the New Drug Application (NDA) submission, which triggered a review extension of three months. Reenie McCarthy, the CEO of Stealth, has indicated that the company remains “confident in the robustness of the NDA package.”
It goes without saying that we are very disappointed in this delay, but we are not disheartened. The regulatory process as it exists today simply does not work for communities like ours for which the need is unmet and incredibly urgent and the number of patients is very small. Our advocacy in Congress and at the FDA has and will continue to progress toward development of regulatory processes that work better for our community and others like ours. This is not a sprint, but a marathon, despite our deep desire that it be different right now.
We wish we had a crystal ball to reassure you that this will work out. What we do have is advice, experience and perspective from experts as well as statistics, and all that makes us cautiously optimistic about this NDA for the use of elamipretide in Barth syndrome. We ask you to continue to join us in solidarity as we do our part to seek a positive outcome in April.
We also want to acknowledge that there are a lot of changes happening rapidly in federal health agencies. While we don’t yet know the impact of these changes, we remain steadfast in our goals to work together for appropriate – and timely – regulatory processes for our community. We have a large group of people from our community going to Rare Disease Week next month to keep our plight actively in the minds of those in Washington, DC. Our continued voicing of our patient perspective is critical both for this NDA as well as for any potential future therapy development efforts for our ultra-rare disease.
We will still host our scheduled community meeting on February 4th and do our best to answer any questions you may have about what this change in date means for you or your family. Register here.
Thank you for your commitment to the Barth community as we keep making #BarthProgress together.
Emily Milligan & Kate McCurdy
