Synthetic Flavanol Gets Orphan Drug Status for Duchenne and Becker Muscular Dystrophy

The Food and Drug Administration (FDA) has granted Orphan Drug designation to EB 002 ((+)-epicatechin; Epirium Bio) for the treatment of Duchenne and Becker muscular dystrophy.

Duchenne and Becker muscular dystrophy (DMD and BMD) are rare, genetic neuromuscular disorders that cause progressive muscle weakness and damage and primarily affect skeletal and cardiac muscle. DMD typically manifests between the ages of 2 and 3 years, while BMD manifests later in life; both disorders are caused by mutations of the dystrophin gene, resulting in absent or defective dystrophin protein.

The novel therapeutic approach of using an orally available, synthetic flavanol to treat these disorders was established in early proof of concept human studies. The Company stated it will initially focus on advancing the treatment in patients with BMD.

“Our initial emphasis on Becker muscular dystrophy stems from our research demonstrating that EB 002 may stimulate mitochondrial biogenesis, leading to the restoration of tissue bioenergetics, while upregulating production of key proteins which may lead to improved muscle structure and function,” said Ransi Somaratne, MD, Epirium’s chief medical officer.

For more information visit epirium.com.