Updated 14 September 2024

Let your voice be heard: Submit Your Written Testimony by September 25th

On September 6th, the U.S. Food & Drug Administration (FDA) announced that the Cardiovascular and Renal Drugs Advisory Committee has scheduled their meeting to review Stealth Biotherapeutic Inc.'s new drug application (NDA) 215244 for elamipretide hydrochloride injection for the treatment of Barth syndrome for October 10th, 2024.

BSF will share a link for you to view the October 10th meeting, and all publicly available meeting materials, will be shared on October 8th. BSF will provide the link and meeting materials on our website once they are publicly posted by FDA. 

At the end of the meeting on October 10th, the Advisory Committee will make a recommendation for or against approval to the FDA. It is important to note that the Committee’s recommendation is NOT the final decision. That decision will be announced by FDA by January 29th, 2025 (also known as the PDUFA date).

The global Barth syndrome community has a valuable role to play in the FDA’s review of elamipretide. As part of their review, Advisory Committee members will have an opportunity to read letters submitted from the BSF community.

We believe it critically important that the FDA and this Advisory Committee hear what it’s like to live with and care for someone with Barth syndrome. That is a story that only you can tell and why the BSF is encouraging you to provide written testimony to the public docket. BSF will hold an essential briefing on this subject on Friday September 13th at 12p ET and will provide a secure Zoom link  for this purpose through our communication channels.  If you would like to attend this meeting reach out to Shelley Bowen

• The deadline for your letter to be submitted AND ensure the Advisory Committee will review it is September 26th, 2024.
• If you would like to submit a letter, we ask that you submit it on September 25th, 2024, which is the day prior to the deadline date

What You Need to Know About Submitting a Letter for Written Comment

• This opportunity to submit a written comment is open to all members of the Barth syndrome community worldwide, not just U.S. residents, but all letters must be submitted in English.
• Letters kept to one page in length are more likely to be read. Letters should address the following:
  • Your Barth syndrome story as a means of conveying concrete examples of the impact of Barth syndrome on day-to-day living including:
    • Physical effects of Barth syndrome
    • Psychological impacts of Barth syndrome
    • Financial impacts of Barth syndrome
    • Caregiving responsibilities
  • Hope for how a drug treatment could protect, maintain, or improve your quality of life
  • Your outlook as you consider drug treatments and weigh potential risks and benefits
• Along with your written letters, individuals and families are encouraged to submit one page of photos to help the Advisory Committee understand the physical impacts of Barth syndrome
• Letters must be submitted on or before September 26th, 2024
• Comments submitted electronically, including attachments, will be posted to the docket unchanged. Your comments will be made public so you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to have posted
  • This includes but is not limited to medical information, your or anyone else’s Social Security number, or confidential business information.
  • If you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on https://www.regulations.gov.
• Comments can be submitted electronically here . You can write directly on the webpage or attach a Word or PDF file with your commentary. For the drop-down question, “What is your comment about?” the question is asking who is submitting the comment – choose “Individual Consumer.”

Please contact Shelley Bowen, Director, Family Services and Advocacy, with any questions you may have regarding submitting a written comment to the Advisory Committee meeting of the FDA.

Visit the Barth Syndrome Foundation advocacy timeline here

Watch September 13th, 2024 Advocacy Essential Training:  How to Submit a Letter to the FDA Docket here

Watch August 1st, 2024 Barth Syndrome Biennial Conference 20204: Lessons from Barth Syndrome Clinical Trials here

Watch May 23rd, 2024 Advocacy Essential Community Briefing: FDA Advocacy Committee here

Learn more about July 18th, 2024 Externally Led Patient Focused Drug Development Meeting here