Ongoing Advocacy Efforts
DRAFT letter from Senator Michael Braun (Indiana) to the U.S. Food and Drug Administration leadership. Senator Braun is currently seeking a Democrat co-lead in the Senate to make this a bi-partisan initiative.
We (BSF) would also like for this to be picked up in a similar bi-partisan manner in the House. This is an issue that affects all Americans who have an ultra-rare condition.
Those fighting with Barth syndrome need you to help them. Click this link to write to the leadership of the FDA to ask for a fair review of elamipretide.
November 1st, 2023 Update: 86 Congressional Meetings since August 2nd, 2023
65 Congressional meetings via Zoom
37 House 14 Democrats/ 23 Republicans
28 Senate 19 Democrats / 9 Republicans
21 In person Congressional meetings
14 House 6 Democrats/ 8 Republicans/ 0 Independents
7 Senate 4 Democrats/ 3 Republicans/ 0 Independents
This timeline is constantly being updated. Please continue to visit this page for updates.
- October 26th, 2023: Emily Milligan, CEO Barth Syndrome Foundation and Dr. Hilary Vernon, PI TAZPOWER Clinical Trial and Director of Barth Syndrome Interdisciplinary Clinic, Kennedy Kreiger Institute invited speakers for Senate Special Committee on Aging, Unlocking Hope: Access to Therapies for People with Rare, Progressive, and Serious Diseases see video here here
- October 12th, 2023: Barth Syndrome Foundation, Director of Family Services and Advocacy, Shelley Bowen, RARECast interview with Daniel Levine. Listen to the podcast here
- October 6th, 2023: Barth Syndrome Foundation, Director of Family Services and Advocacy, Shelley Bowen and CEO Stealth BioTherapeutics CEO, Reenie McCarthy guests on MitoAction Expert Series webinar. Watch the webinar here.
- September 13th, 2023: Barth Syndrome Foundation Executive Director Emily Milligan, and Director of Family Services and Advocacy, Shelley Bowen are interviewed for Mito Action Energy in Action Podcast with Marcy Young. Listen to the podcast here
- September 12th, 2023: Barth Syndrome Foundation launches petition asking officials of the Food and Drug Administration to fairly review the only potential treatment for Barth syndrome featuring the story of one child who may be severely impacted by no longer having access to elamipretide. Sign the petition here
- September 7th, 2023: Impasse at U.S. Food and Drug Administration could mean Stealth Biotherapeutics abandons Barth syndrome treatment. Read PINK SHEET Citeline Regulatory here
- September 6th, 2023: Boston Business Journal publishes article about uncertain future in obtaining U.S. Food and Drug Administration approval of elamipretide as a therapy for Barth syndrome patients. Read Boston Business Journal article here
- September 1st, 2023: Barth Syndrome Foundation holds workshop, “Preparing for your Legislative Meeting,” with Kevin Freiert (CEO, Salem Oaks). Watch webinar here
- August 25th, 2023: Barth Syndrome Foundation holds webinar, “Advocacy in Action: Why you Matter,” with Katie Landes (KPM Group DC), Jake Chebowsky (Senator Braun, R-IN) and Jackie Weinrich (US Representative Matsui, D-CA-07). Watch webinar here
- August 17th, 2023: Barth Syndrome Foundation holds webinar, “Special Meeting on Elamipretide,” with Stealth BioTherapeutics, Drs. Todd Cade and Hilary Vernon, and affected families. Watch webinar here
- August 1st, 2023: Barth Syndrome Foundation launches Congressional advocacy campaign for equitable, fair, and appropriate review of elamipretide by the Food and Drug Administration.
- July 27th, 2023: Tiny biotech says its troubles make case for ultra-rare Food and Drug Administration pathway; highlights challenges to get therapy for Barth syndrome approved by the Food and Drug Administration. Read BIOCENTURY article here
- Late June, 2023: Barth Syndrome Foundation joins Stealth BioTherapeutics for a Type A meeting with the Division of Cardiology and Nephrology at FDA.
- June 16th, 2023: Temporal evolution of the heart failure phenotype in Barth syndrome published with evidence that left ventricular cardiac volumes decline with advancing age, suggestive of pathological cardiac remodeling. Read Future Cardiology article here
- April 5th, 2023: Rep. Paul Tonko [D-NY-20] Sponsors House Resolution H.Res.276 designating April 5th, 2023 as Barth Syndrome Awareness Day. Read full story here
- March 17, 2023: Optimization of positive investigational outcomes in Barth syndrome using a combination of placebo-controlled, extended open-label, and natural history comparison studies.
Data from close-out of the open-label trial demonstrates (i) a highly significant >40% improvement in heart function (specifically, left ventricular stroke volume) and (ii) a highly significant improvement in the biomarker that is diagnostic for Barth syndrome. Improvement in exercise tolerance (>25% improvement from baseline on 6MWT) and strength (>30% improvement from baseline on muscle strength) were maintained. Read American College of Medical Genetics Meeting abstract here
- January 27, 2023: Global Genes RAREcast Daniel Levine interviews Reenie McCarthy, CEO of Stealth BioTherapeutics, about the challenges the company has faced in seeking FDA approval for elamipretide, therapy for Barth syndrome. Reenie speaks to the lack of consistency within the agency, and why this could have a chilling effect on the development of ultra-rare disease therapies if left unaddressed. Listen to podcast here
- September 2, 2022: Natural history comparison study to access the efficacy of elamipretide in patients with Barth syndrome which meets endpoints with high statistical significance. Read Orphanet Journal of Rare Diseases article here
- August, 2022: Barth Syndrome Foundation joined Stealth BioTherapeutics for a pre-NDA meeting with the Division of Cardiology and Nephrology
- August 2, 2022: A second physician-led letter signed by 25 key opinion leaders was submitted to FDA, again requesting expedited access to elamipretide for patients living with Barth syndrome. Download letter here
- August 1, 2022: Longitudinal observational study of cardiac outcome risk factor prediction in children, adolescents and adults with Barth syndrome demonstrating expected decline of left ventricular volumes for teens and adult patients in the natural history. Read Pediatric Cardiology article here
- July 29, 2022: The Barth Syndrome Foundation (BSF), including BSF representatives and key clinical disease leaders, held a workshop with Dr. Norman Stockbridge, Director of the Division of Cardiology and Nephrology in the Center for Drug Evaluation and Research at the Food and Drug Administration. Over 30 representatives from various divisions of the Food and Drug Administration attended this meeting. The stated purpose of this workshop was “to identify, through informed discussions, executable regulatory pathways for Barth syndrome therapy development and establish regulatory paths to approval.” It was a rare opportunity to further educate the FDA about disease-specific details of Barth syndrome and to initiate work that we hope ultimately will lead to consensus about possible paths forward for future approved treatments for our ultra-rare disease. Read report here
- June 28, 2022: Barth Syndrome Foundation ambassadors meet with legislators from their home states to advocate for improved U.S. Food and Drug Administration review processes for ultrarare indications, like Barth syndrome. Read more here
- June 14, 2022: The Barth Syndrome Foundation responds to the U.S. Food and Drug Administration granting pre-NDA meeting for elamipretide to Stealth Biotherapeutics. Read more here
- March 7, 2022: Barth Syndrome Foundation demands transparency of patient input into regulatory decision making. Barth Syndrome Foundation’s executive Director, Emily Milligan speaks at 7th annual Biopharma Congress about the lack of transparency in which the U.S. Food and Drug Administration is incorporating patients’ perspectives into the agency’s review protocols. Read article here
Emily Milligan is interviewed for PINK SHEET feature on Patient-Focused Drug Development Meetings. Read PINK SHEET Citeline Regulatory article here
- February 22, 2022: The Barth Syndrome Foundation requests a meeting with the U.S. Food and Drug Administration to highlight insurmountable statistical hurdles required for ultra-rare conditions such as Barth syndrome. Read more here
- November, 2021: Barth Syndrome Foundation joins Stealth BioTherapeutics at Type A meeting with Division of Cardiology and Nephrology at FDA.
- October 21, 2021: Barth Syndrome Foundation Board Chair, Kate McCurdy, to the U.S. Food and Drug Administration refusal to file Stealth BioTherapeutics' NDA for elamipretide. Read letter here
- October 20, 2021: U.S. Food and Drug Administration refuses to file Stealth Biotherapeutics New Drug Application for elamipretide for treatment of Barth syndrome. Read Med City News article here
- September 20, 2021: Business of Rare Fireside Chat with Emily Milligan, Barth Syndrome Foundation and Reenie McCarthy, Stealth BioTherapeutics. Watch here
- September 15, 2021: Rare Disease Research: A Prescription event sponsored by STAT News features interviews with Walker Burger, a TAZPOWER study participant, and Jay Randell, the father of two TAZPOWER study participants, and a panel discussion Ultra Rare, Ultra Difficult with Ed Silverman, Pharmalot, Frank Sasinowski, Hyman Phelps, and Reenie McCarthy, Stealth BioTherapeutics. See program here
- September 14, 2021: In a STAT News opinion piece entitled Aduhelm backlash threatens to reverse progress in U.S. Food and Drug Administration’s reviews of rare and ultra-rare disease drugs, Emily Milligan, and Kate McCurdy point out inconsistencies in the U.S. Food and Drug Administration’s approach to operationalizing the 21st Century Cures Act stand to treat individuals with Barth syndrome qualitatively differently from those with other diseases, such as Alzheimer’s, and points to a critical need for consistent application of patient perspectives in the review process across agency divisions and regulators. Read STAT News article here
- September 13, 2021: Abe’s parents share their personal experience and hope they hold for therapy to help their son. Watch video here
- September 2, 2021: Stealth elamipretide New Drug Application tests frontiers of U.S. Food and Drug Administration’s flexibility in rare diseases. Read PINK SHEET article here
- August 24, 2021: Stealth BioTherapeutics submits elamipretide New Drug Application to U.S. Food and Drug Administration for treatment of Barth syndrome. See Newswire article here
- March 3, 2021: The Barth Syndrome Foundation held a Listening Session with 28 participants from various areas within the U.S. Food and Drug Administration to discuss caregiver perspectives on tolerance for risk of uncertainty of treatment benefit. Read Summary here
- Spring 2021: Review Division #4: Investigational New Drug Application transferred to Division of Cardiology and Nephrology.
- February, 2021: The Barth Syndrome Foundation joins Stealth BioTherapeutics for a Type C meeting with the Division of Cardiology and Nephrology at FDA.
- December 3, 2020: 4,256 individuals signed a petition to Stealth BioTherapeutics and the U.S. Food and Drug Administration requesting that Stealth BioTherapuetics submit a New Drug Application and for the U.S. Food and Drug Administration file and review the New Drug Application for elamipretide. Read 2020 Petition Summary here
- November 11, 2020: Dr. John Lynn Jefferies plus  key opinion leaders submit a physician-led letter to FDA requesting expedited access to elamipretide for the treatment of Barth syndrome. See article and read letter here
- September 18, 2020: Barth Syndrome Foundation Community Roundtable session featuring TAZPOWER clinical trial participant Walker Burger’s testimony about his experience while taking elamipretide. Watch video here
- March 2020: Barth Syndrome Foundation joins Stealth BioTherapeutics for a Type C meeting with the Division of Rare Disease and Medical Genetics to discuss positive Phase 3 natural history control study data
- Winter 2020 Review Division #3: U.S. Food and Drug Administration, Division of Rare Disease, and Medical Genetics (reorganized division of Gastroenterology and Inborn Errors of Metabolism) declined to review the Phase 3 data.
- February 18, 2020: Stealth BioTherapeutics announces positive data from Phase 3 natural history control study. Read here
- February 2020: A Bayesian Analysis to Determine the Prevalence of Barth Syndrome in the Pediatric Population, Published in The Journal of Pediatrics estimating the prevalence of Barth syndrome to be 1:1,000,000 males which translates to approximately 150 males living with Barth syndrome in the United States. At the time of this publication the Barth Syndrome Foundation is aware of 125 people diagnosed with Barth syndrome in the United States. Read The Journal of Pediatrics article here
- Spring 2019: Barth Syndrome Foundation joined Stealth BioTherapeutics for a Type B end-of-Phase 2 meeting with the Division of Neurology Products at the FDA.
Following this meeting between Stealth Biotherapeutics and the Division of Neurology Products (Review Division #1), the investigational new drug application was transferred to the Division of Gastroenterology and Inborn Errors of Metabolism (Review Division #2).
- April 25, 2019: Barth Syndrome Foundation met with 20 U.S. Food and Drug Administration officials in Silver Spring, MD, including senior representatives from the Division of Neurology Products, the Division of Gastroenterology and Inborn Errors of Metabolism, and the Division of Cardiology and Nephrology to explain the ultra-rare nature of the disease and its severe impact on patients’ lives.
- March 20, 2019: Barth Syndrome Foundation travel to Silver Spring Maryland to present Voice of the Patient written summary to representatives of the U.S. Food and Drug Administration. Read Voice of the Patient report here
U.S. Food and Drug Administration releases March 2019 Rare Diseases: Natural History Studies for Drug Development. Read here
- February 11, 2019: Functional exercise capacity, strength, balance, and motion reaction time in Barth syndrome published by researchers at Kennedy Krieger Institute, Barth Syndrome Clinic. Read Orphanet Journal of Rare Diseases article here
2017 - 2018
- July 20, 2018: Summarized findings from the Barth registry interviews: Key points for moving forward; Anthony Aiudi, PharmD, Stealth Biotherapeutics, Newton, MA. Barth Syndrome International Conference. See recording here
- July 18, 2018: Barth Syndrome Foundation host Externally Led Patient Focused Drug Development Meeting. View full recording here
- May 18, 2018: Bob Buckley shares his hopes for a future therapy to allow him to just be able to drive his car again. Bob passed away in September of 2021 before that simple request could be delivered. View recording here
- 2017 - 2018: Phase 2/3 conducted at Johns Hopkins. Trial screened 16 individuals and enrolled 12 Barth syndrome patients. Read Genetics in Medicine article here
- May 23, 2017: Exploring the sign and symptom experience of Barth syndrome in adult and adolescent populations. Stokes J, Aiudi A, Mazar I, Elliott M, Dillard S, Ollis S, Love E, Shields AL, Gwaltney C. 22nd Annual International Meeting of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), Boston, MA USA, 20-24 May 2017 See abstract here
- February 4, 2016: New targets for monitoring and therapy in Barth syndrome published. Read Genetics in Medicine article here
- July 19, 2016: Stealth BioTherapeutics in collaboration with the Barth Syndrome Foundation develops Barth syndrome symptom assessment patient reported outcome tool informed by concept elicitation interviews with pediatric and adult affected individuals and/or their caregivers and evaluated in cognitive debriefing interviews to assess relevance and readability. This endeavor helped identify the impact of fatigue as a central concern for affected individuals. Read Orphanet Journal of Rare Disease article here.
- April 9, 2015: Barth syndrome Foundation reached out to Stealth to determine if people with Barth syndrome would be eligible to participate in MMPOWER (bendavia /now elamiprtide) clinical trial
- October 4, 2013 Webinar Introducing Bendavia (now elamipretide) as a possible therapy for people with mitochondrial disease hosted by MitoAction see summary of webinar here