Barth Syndrome Foundation, UMDF, and MitoAction leadership were recently notified by Stealth BioTherapeutics that FDA has refused their request to reconsider the elamipretide new drug application (NDA). This refusal to reconsider comes despite a positive Type A meeting where FDA reviewers signaled that Stealth should proceed with a formal request for reconsideration. Instead, FDA has requested that Stealth BioTherapeutics resubmit their NDA, for the third time, using the accelerated approval pathway but under the longer of two resubmission timeline options. There is a lot we don’t know, and information is still emerging, but we urgently feel that the community should know what we have learned thus far, in as real time as is feasible.

Here is what we know:

• FDA asked Stealth to resubmit their NDA under the accelerated approval pathway but with a 6-month review timeline instead of a shorter 2-month period.
  • This 6-month timeline does not include the 30-day process FDA needs to accept the NDA for review.
• Stealth’s investors have said they cannot, under the current circumstances, continue to guarantee funding for the company. These unanticipated and continual delays have reduced their confidence in future expediency by FDA.
• All issues related to manufacturing that were mentioned in Stealth’s complete response letter (CRL) have been addressed and should not impede reconsideration.
• Barring a last-minute intervention by FDA or HHS leadership, the Expanded Access Program (EAP) through which all individuals are currently receiving elamipretide will most likely end.

Here is what we do not know:

• We do not know specifically when EAP access to elamipretide will end but it could be as early as September.
• We do not know if Stealth will proceed with resubmission of their NDA under this accelerated approval pathway. Without approval now, reconsideration, or a written commitment from FDA for a shorter 2-month review period, Stealth’s investors are unlikely to consider further investment.

What are the asks?

• On social media, ask FDA to approve now or approve fast to avoid disrupting elamipretide access. All manufacturing technicalities have been addressed. A 6- month review period is too long and it is Barth-affected individuals who will pay the ultimate price.
  • Hashtags to use and people to tag: #approveelamipretide #barthprogress #fdaactnow #barthsyndrome #raredisease The White House Rare Disease Legislative Advocates S. Department of Health and Human Services U.S. Food and Drug Administration
    
    
• Use your connections—if you or loved ones have means of reaching those in power (FDA Commissioner Makary, HHS Secretary Robert F. Kennedy Jr. or President Donald Trump), now is the time!
  • Convey the following messages:
    • Barth syndrome is a life-limiting, ultra-rare, mitochondrial disease with no approved treatments
    • What should have been a 6-month FDA review process for elamipretide is entering its 19^th month and, in that time, the Sponsor (Stealth) has utilized all of its financial runway and cannot afford further work—which means Barth patients will lose access.
    • Many mitochondrial disease patients with other diagnoses have also benefited from elamipretide and now are at risk for losing access as well.
    • FDA’s failure to use the regulatory flexibility granted by Congress is causing ALL Barth-affected individuals and those on expanded access to lose access to the only drug shown to improve symptoms associated with Barth syndrome and other mitochondrial diseases.
    • We are pleading with those in power to intervene and overturn the CRL or, if absolutely necessary, grant a 2-month approval review. Individuals with Barth syndrome and the broader mitochondrial disease community are being denied the right to try.

We know that each member of this community has advocated tirelessly on this journey and that this is news is devastating. We will continue to share updates here and, where appropriate, on social media. We are pushing for last-minute meetings with key decision-makers to advocate and are pursuing every remaining lead.

This news does not define the future of our community. Every challenge we face only strengthens our resolve to keep fighting, keep pushing, and keep showing up—we will never give up. We will continue to stand together, raise our voices, and advocate for the therapies our loved ones need and deserve.