The Food & Drug Administration (FDA) has given ray of hope to Sarepta Therapeutics by agreeing to consider its experimental drug for Duchenne muscular dystrophy for accelerated approval.
Sarept’s eteplirsen is a new intravenous drug known to stabilize the symptoms of a rare and fatal form of muscular dystrophy known as Duchenne.
Expressing confidence in positive FDA decision, Sarepta Chief Executive Officer Chris Garabedian said, “We’re very pleased we received this guidance from the FDA. It provides us all the information we need to move our program forward. We’re really excited about this news and we think we will be in good shape to submit our [new drug approval application] by the end of the year.”
Sarepta Chief Medical Officer Ed Kaye said he would move expeditiously to begin the additional trials.
The drug ‘Eteplirsen’ helps in checking a genetic mutation that affects 13 percent of Duchenne patients. Experts believe, FDA’s approval would open the door to development of other drugs to treat Duchenne boys with other genetic mutations.
The drug maker, as per the discussion with its investors, will conduct two additional studies approval that could bring the drug to market by summer 2015.
The firm has also decided to use a newer and more precise method of measuring dystrophin, the essential muscle protein that Duchenne boys lack.
Meanwhile, the federal agency has expressed concerns over the authenticity as far as size and scope of clinical trials are concerned.
After the decision, the shares of Sarepta (SRPT) jumoed $11.34, or 46%, to $35.74.