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

Global Leigh Syndrome Treatment Market, By Disease Type (Mitochondrial Complex I Deficiency, Mitochondrial Complex III Deficiency, Mitochondrial Complex IV Deficiency , LSAD, Others), By Drug Type (Vitamin Supplements, Antioxidants, Cannabidiol, Anticonvulsants, Others), By Age Group (Infants, Teenagers and Adults), By Distribution Channel (Hospital Pharmacies, Retail pharmacies, Online Pharmacies), By Geography (North America, Europe, Asia Pacific, Latin America, Middle East and Africa)

Recent Developments

Product Designation

On December 14, 2023, Abliva AB, a clinical-stage company developing medicines for the treatment of rare and severe primary mitochondrial disease, announced that the European Commission had granted orphan designation for the company’s drug candidate NV354 for the treatment of Leigh syndrome, facilitating its clinical development in neurological mitochondrial disease.
On September 14, 2023, Cannabidiol had been given orphan drug designation by the European Medicines Agency (EMA) to be used in the treatment of Leigh syndrome, a rare mitochondrial disease. The decision comes after a petition made by researchers from the Mitochondrial Neuropatology research group at the UAB Institut de Neurociènces (INc-UAB) Emma Puighermanal, Albert Quintana and Elisenda Sanz.

Business Development Activities by Market Players

On June 29, 2023, PTC Therapeutics, Inc., a pharmaceutical company, announced that the MIT-E trial of vatiquinone for the treatment of mitochondrial disease associated seizures (MDAS) failed to achieve its primary endpoint of reduction in observable motor seizures. The study showed evidence of treatment effect in reducing seizure frequency in the overall study population and in the largest subgroup of children with Leigh syndrome, in whom benefit was also observed in the key secondary endpoints of occurrence of status epilepticus and disease-related hospitalizations.
On June 5, 2023, Cure Mito Foundation, a parent-led organization dedicated to advancing research and treatments for Leigh syndrome, launched the first-of-its-kind online resource about Leigh syndrome, the most common type of pediatric mitochondrial disease. The free resource, “About Leigh Syndrome,” serves as a central place where patients, caregivers, and doctors can find information on Leigh syndrome, including its symptoms, diagnosis, treatment options, and others.