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Universitat Autònoma de Barcelona

EMA gives cannabidiol orphan drug designation for the treatment of Leigh syndrome

14 Sep 2023
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Cannabidiol has 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 Neurociències (INc-UAB) Emma Puighermanal, Albert Quintana and Elisenda Sanz.

L'equip de recerca a l'INc
The research team: Elisenda Sanz (left), Albert Quintana and Emma Puighermanal-Puigvert.

Rare diseases are those that have an incidence equal to or less than 5 cases per 10,000 people. They are so rare that, despite their severity, they are often ignored by the pharmaceutical industry. To facilitate the development and authorisation of drugs for rare diseases, regulatory agencies can grant certain medicines an orphan drug designation (ODD), which then qualifies them for several incentives.

Leigh syndrome is a very severe neurodegenerative mitochondrial disease affecting the paediatric population. Because mitochondria do not work well, patients have problems in many organs and systems, but mainly in their muscles and nervous system. The causes of the disease are genetic and there are currently no approved treatments.

In an extensive preclinical study combining the use of animal models of this disease with patient-derived cells, researchers of the INc-UAB have shown that cannabidiol, a chemical compound from the cannabis plant, exerts multiple beneficial effects. Among them, they found a decrease in the production of oxidative stress and inflammation, an improvement in social and motor problems, a reduction in epilepsy and an increase in life expectancy in Leigh syndrome mouse models.

The leaders of this project, Dr Emma Puighermanal and Dr Albert Quintana, expect to be able to conduct a clinical trial soon about the potential use of cannabidiol as a treatment for Leigh syndrome. To facilitate the development of a treatment, they have just obtained an orphan drug designation by the European Medicines Agency, which entails several benefits such as scientific advice and assistance in protocols, market exclusivity and reduced development costs, among others.

For more information: Union Register of medicinal products https://ec.europa.eu/health/documents/community-register/html/o2800.htm