The FDA has refused Stealth BioTherapeutics’ request to reconsider its application for elamipretide—despite earlier indications from FDA reviewers that such a request was appropriate. Instead, the FDA has instructed Stealth to resubmit under the accelerated approval pathway with a 6-month review timeline, the longer of two options. This delay, coupled with financial constraints facing Stealth, puts the future of elamipretide—and the Expanded Access Program (EAP) through which patients currently receive it—at serious risk. Without immediate action or a shortened review, access could end as soon as September, leaving individuals with Barth syndrome and other mitochondrial diseases without the only treatment shown to improve symptoms.

We are calling on the community to advocate by participating in GO Grassroots for Elamipretide. We have outlined a week of ways to participate in advocacy from anywhere, from August 18 through 22, to convey the urgency and to keep elamipretide at the forefront. Every voice matters—together, we can push for the urgent action needed to preserve access to this critical therapy.

If you have Barth syndrome or a mitochondrial condition, or know someone who does, we encourage you to participate in our GO Grassroots for Elamipretide effort by clicking any or all of the following ways: