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The Barth Syndrome Foundation (BSF) is deeply disappointed by the US Food and Drug Administration’s (FDA) refusal to file Stealth BioTherapeutics’ (Stealth) submission of a New Drug Application (NDA) for elamipretide as a treatment for those living with Barth syndrome.

While the decision is a setback for the Barth syndrome community, we recognize the tremendous, collective efforts the BSF community has conducted in advocating for access to therapies (click on any of the blue text to learn more):

  • July 2018: BSF hosted for the first externally led Patient Focused Drug Development meeting outside of the metro DC area 
  • March 2019: Submitted the Voice of the Patient Summary Report to the FDA 
  • March 2019: Spoke to the FDA at a Barth syndrome listening session at FDA headquarters in Maryland 
  • November 2020: Submitted to the FDA a community petition signed by more than 4200 individuals advocating for patient access to elamipretide (click to learn more)
  • November 2020: Supported a key opinion letter signed by 26 clinical experts addressed to the FDA supporting the use of elamipretide in Barth syndrome patients
  • March 2021: Invited by the FDA to a listening session (virtual) to discuss the Barth syndrome community’s tolerance of clinical benefit uncertainty for therapies.

We commend Stealth for listening to Barth syndrome patients’ and families’ urgent appeal to submit the NDA, which marked a milestone in the journey to a treatment for Barth syndrome. BSF’s commitment will not waiver as we continue to appeal to both Stealth and the FDA to create a pathway for Barth syndrome patients to commercially access elamipretide.

Barth syndrome is a life-threatening and life-limiting genetic disease and presently one of the more than 6,500 rare diseases without an approved treatment. While our disease is ultra-rare, this should not preclude Barth syndrome patients from being able to access therapies and treatments. We at BSF remain steadfast in our commitment to advocate that the FDA actively incorporate patient perspectives into regulatory evaluations now and in the future.


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