News
Article
Stealth Announces Yet Another Resubmission of Barth Syndrome Treatment NDA
Author(s):
Key Takeaways
- Stealth BioTherapeutics is resubmitting its NDA for elamipretide after resolving FDA manufacturing concerns, seeking expedited approval for Barth Syndrome treatment.
- The small patient population for Barth Syndrome complicates clinical trials, making it difficult to obtain statistically conclusive results.
The disease doesn’t have any FDA approved treatments, leaving families desperate for FDA to approve the treatment.
Stealth announced that it has resubmitted its NDA for elamipretide once again after resolving concerns cited by FDA in May.
Stock.adobe.com
One drug’s complicated path to approval demonstrates the necessity for an efficient drug approval process at FDA.
Stealth BioTherapeutics announced that it is resubmitting its new drug application (NDA) for its Barth Syndrome treatment elamipretide.1 The drug is a first-in0class mitochondrial targeted therapeutic that is also in development for primary mitochondrial myopathy and dry age-related macular degeneration.
Why did FDA ask Stealth to resubmit its NDA for its Barth syndrome treatment?
In a press release, Stealth’s CEO discussed resubmitting the NDA, saying, “We appreciate FDA’s recent recognition of the seriousness of Barth syndrome and the unmet need for potential therapies, and its commitment to complete its review of our NDA resubmission as expeditiously as possible. With FDA’s confirmation that the manufacturing observations cited in the CR have been resolved, and the progress we have made to align on post-marketing trial design, we believe that all concerns raised by the FDA have been addressed.”
In May 2025, FDA issued a complete response (CR) to Stealth’s NDA, citing concerns with a third-party manufacturing facility that were raised during a surprise inspection. The CR recommended resubmitting the NDA for accelerated approval. Later, in July, FDA confirmed that the facility was in full CGMP compliance, and no further corrective action was required by Stealth.
While the agency has suggested the new submission would be considered a Class 2 resubmission (which gives the review proves a six-month timeline), Stealth is requesting that FDA consider the resubmission Class 1, which would decrease the timeline to two months. Stealth argues that there is a desperate need for the approval, as there is no FDA-approved treatment for Barth syndrome.
Kate McCurdy, co-founder of the Barth Syndrome Foundation, spoke with NBC News about the situation with elamipretide.2 According to her, testing treatments for conditions like Barth syndrome is hard due to the incredibly small patient population. Barth impacts about one-in-1,000,000 males worldwide, and children suffering from the condition have incredibly short life expectancies.
This makes collecting data from clinical trials very difficult, as the small population size makes it impossible to get results that are conclusive beyond a statistical doubt.
The report also cites changes in leadership at FDA as another obstacle. After the results of the 2024 presidential election, a new administration took office with a stated goal of making big changes to government agencies. HHS and FDA were not immune, and key leadership roles changed, and staff sizes were reduced.
A former FDA official spoke with NBC under the cover of anonymity, saying, “Those who have taken over are not as familiar, or perhaps it’d be more appropriate to say they’re inexperienced with how one does drug approvals, how one drives things forward, and they’re also somewhat fearful of the current environment. And I think that’s led to an adverse outcome for some of these rare disease products.”
It is important to note, however, that Stealth’s difficulties began under the previous administration. However, with the changes in leadership, the new officials in key roles may be unfamiliar with the drug and its journey to approval.
As the anonymous official described it, elamipretide has become a “hot potato.” For families living with the disease, this delay is more than just frustrating. A delay in receiving treatment can literally mean life or death for the young victims of the syndrome.
Sources
- Stealth BioTherapeutics resubmits new drug application for elamipretide for the treatment of Barth Syndrome. Stealth BioTherapeutics. August 18, 2025. https://stealthbt.com/stealth-biotherapeutics-resubmits-new-drug-application-for-elamipretide-for-the-treatment-of-barth-syndrome/
- Time is running out for kids with a rare disease, parents say, unless the FDA acts. NBC News. August 18, 2025. https://www.nbcnews.com/health/kids-health/barth-syndrome-children-fda-elamipretide-drug-rare-disease-rcna224858
Newsletter
Lead with insight with the Pharmaceutical Executive newsletter, featuring strategic analysis, leadership trends, and market intelligence for biopharma decision-makers.
