The designation supports the development of experimental treatments for rare diseases (those that affect fewer than 200,000 people in the U.S.). It also comes with certain benefits, including financial incentives for clinical development and commercialization, special fee exemption, as well as exclusivity in the U.S. market for seven years.
Epirium is planning to focus first on developing EB 002 as an oral treatment for people with Becker MD. According to the company, existing data are promising. Specifically, results suggested that EB 002 may be able to promote the formation of new mitochondria — the cell compartments responsible for the production of energy — and possibly stimulate the production of proteins needed for muscle health.
“Our initial emphasis on Becker muscular dystrophy stems from our research demonstrating that EB 002 may stimulate mitochondrial biogenesis [formation of new mitochondria], leading to the restoration of tissue bioenergetics, while upregulating [promoting] production of key proteins which may lead to improved muscle structure and function,” Ransi Somaratne, MD, chief medical officer of Epirium, said in a press release.
Russell Cox, the company’s CEO, added: “We are delighted that the data presented on EB 002 supported the FDA’s grant of an orphan-drug designation. This is an important milestone for the development of our potential treatment for Becker muscular dystrophy patients, for whom there are no specific treatment options.”
Like Duchenne, Becker is a form of muscular dystrophy caused by mutations in the dystrophin (DMD) gene. Although patients with Becker are still able to produce dystrophin, the protein either malfunctions or its levels are insufficient, leading to muscle weakness and heart problems.
Becker is considered a rare disease, with an estimated prevalence of one in 18,000 to one in 30,000 male births. Its progression is typically slower than seen in people with Duchenne.
“Our hope is that this designation along with our robust clinical development program will lead to potential new therapies in diseases where they are urgently needed,” Cox said.
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