BARTH SYNDROME TREATMENT MARKET SIZE AND SHARE ANALYSIS - GROWTH TRENDS AND FORECASTS (2024-2031)

Barth Syndrome Treatment Market, By Therapeutic Approaches (Enzyme Replacement Therapy, Gene Therapy, Supportive Care, Stem Cell Therapy, and Others (Small Molecule Drugs)), By Drug Classes (Cardiolipin Precursors, Antioxidants, Immunomodulators, Antibiotics, Dietary Supplements, and Others), By Route of Administration (Oral, Intravenous, and Others (Intramuscular and Topical)), By End User (Hospitals, Specialty Clinics, Research Institutions, and Others), By Geography (North America, Latin America, Asia Pacific, Europe, Middle East, and Africa)

On April 2024, Stealth BioTherapeutics announced that the FDA had accepted its New Drug Application (NDA) for elamipretide, intended to treat Barth syndrome. The NDA is backed by positive results from the SPIBA-001 Phase 3 study and additional data from the TAZPOWER Part 2 trial. Elamipretide has previously received Fast Track, Orphan Drug, and Rare Pediatric Disease designations.
In May 2023, Pharmanovia expanded its cardiology portfolio by acquiring licensing rights to elamipretide for Barth syndrome in Europe and MENA through an agreement with Stealth BioTherapeutics. The partnership aims to finalize studies showing elamipretide's potential to improve the quality of life for those with Barth syndrome, a rare and serious condition. Promising results have already been seen in ongoing trials, and the drug has received Orphan Drug designations from both the FDA and EMA, highlighting the unmet need in this area.