Donna Cowan, Associate Director Expanded Access Programs and Registry at Stealth BioTherapeutics, discusses expanded access programs and the company’s drug candidate, elamipretide, for Barth syndrome.

 

 

Expanded Access Programs

Expanded access programs, also known as compassionate use programs, provide an opportunity for patients diagnosed with rare diseases with no approved therapies. Through these programs, patients may be approved to use therapies that have not yet been approved by the U.S. Food and Drug Administration (FDA) or other regulatory bodies outside the United States. Access to these therapies come with no cost to the patient when accepted into an expanded access program. As Ms. Cowan explains, typically, patients enrolled in these programs are those that do not meet the requirements to participate in a clinical trial.

Barth Syndrome Drug Candidate

Barth syndrome is a metabolic and neuromuscular disorder that primarily affects the heart, immune system, muscles, and growth. It is caused by genetic changes in the TAZ gene and occurs almost exclusively in males. The main characteristics of the condition include:

  • Abnormalities of heart and skeletal muscle
  • Low levels of neutrophils that help to fight bacterial infections
  • Growth retardation, potentially leading to short stature

Other signs and symptoms may include increased levels of certain organic acids in the urine and blood, and increased thickness of the left ventricle of the heart due to endocardial fibroelastosis, which can cause potential heart issues.

Elamipretide is an aromatic-cationic, cell-permeable tetrapeptide. By targeting the mitochondria via cardiolipin, the therapy improves energetics and decreases reactive oxygen species, stabilizing the mitochondrial membrane and cytochrome.

The FDA has recently accepted the New Drug Application (NDA) for elamipretide for the treatment of Barth syndrome. The NDA follows positive data from the SPIBA-001 Phase 3 Natural History Control study as well as safety and efficacy data from the TAZPOWER Part 2 trial.

To learn more, visit https://stealthbt.com/stealth-biotherapeutics-announces-fda-acceptance-of-new-drug-application-for-elamipretide-for-the-treatment-of-barth-syndrome/

To learn more about Barth syndrome and other rare metabolic disorders, visit https://checkrare.com/diseases/metabolic-disorders/