Developer of novel RNA-targeted therapeutics, Sarepta Therapeutics, recently announced the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System Drugs Advisory Committee has given a tentative schedule after its review of the company’s filed New Drug Application (NDA) for the Duchenne muscular dystrophy (DMD) product eteplirsen. The committee initially set the review for January 22, 2016. However, there has yet to be a confirmed meeting date for the advisory committee.
Eteplirsen, the company’s lead candidate for DMD, is formulated to address the disease at its root cause by stimulating the production of a functional dystrophin protein. Previously completed clinical tests were able to demonstrate eteplirsen’s ability to restore protein expression while maintaining a favorable safety and tolerability profile.
“We have been verbally informed by the FDA that we have a tentative date of January 22, 2016, for our advisory committee review of eteplirsen and our PDUFA date of February 26, 2016, remains unchanged,” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “We look forward to discussing the efficacy and safety data included in our NDA submission for eteplirsen with the committee, with the ultimate goal of bringing treatment to more patients with Duchenne.”
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is the most common inherited muscle disease, leading to severe disability and death in young men, caused by the progressive degeneration of striated muscles aggravated by sterile inflammation. The pleiotropic effects of this genetic condition also include cognitive and behavioral impairments, and low bone density. Worldwide, DMD affects nearly one in every 3,500 boys.
In a recent study published in the journal PLOS Medicine, a team of researchers from the University of Portsmouth, U.K., led by Professor Darek Gorecki, were able to block a protein called P2RX7, involved in Duchenne muscular dystrophy (DMD). The results established that a specific treatment can improve both short- and long-term muscle function, and also correct cognitive impairment and bone loss in a DMD mice model. Although the experiments are still at an early stage and no tests have been performed in humans, these findings could lead to a promising treatment for patients with DMD.
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