Serapta announced that the FDA will continue to review the drug past the PDUFA date and that it hopes to finish its work in a timely manner.
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A rejection would be devastating for the many boys with Duchenne and could cripple Sarepta.
However, families of patients with DMD, who generally don’t live past the age of 30, have been lobbying hard for some treatment to come on the market, since now none are available. Sarepta Therapeutics, Inc. was founded in 1980 and is headquartered in Cambridge, Massachusetts. For more information, please visit us at www.sarepta.com.
Eteplirsen is created to address the underlying cause of DMD by restoring the dystrophin messenger RNA (mRNA) reading frame, thereby enabling the production of a shorter, functional form of the dystrophin protein. DMD kills most patients by age 25 as their muscles fail, destroying their ability to walk and, eventually, to breathe. Eterplirsen is targeted at a smaller subset of roughly 13% of the wider DMD patient population. Promoting the synthesis of a shorter dystrophin protein is meant to slow the decline of ambulation and mobility seen in DMD patients. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. FDA is slated to make its decision on whether to approve eteplirsen by this coming Thursday.
Separately, TheStreet Ratings team rates the stock as a “sell” with a ratings score of D-.
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The FDA’s decision will be a critical one for the company: At the end of March Sarepta had $141 million in cash and equivalents, down $63 million from three months earlier. We encourage investors and potential investors to consult our website regularly for important information about us.
