Biotechnology company Stealth BioTherapeutics (NASDAQ:MITO) on Tuesday announced that it has received US Food and Drug Administration (FDA) Rare Pediatric Disease (RPD) designation for elamipretide for the treatment of Barth syndrome, a very rare genetic condition.

Barth syndrome is said to be characterised by cardiac abnormalities often leading to heart failure and reduced life expectancy, recurrent infections, muscle weakness and delayed growth. Barth syndrome occurs almost exclusively in males.

Following the US FDA approval of elamipretide for Barth syndrome, the company is eligible for a voucher that can be used to obtain priority review for a subsequent human drug application upon meeting relevant statutory requirements associated with the RPD program.

Mitochondria are the body's main source of energy production and are critical for normal organ function. Dysfunctional mitochondria characterize a number of rare genetic diseases and are also involved in many common age-related diseases, The company believes elamipretide, has the potential to treat both rare metabolic cardiomyopathies, including Barth, as well as ophthalmic diseases.