Shares of Sarepta Therapeutics rose after the company said that it will apply again to the U.S. Food and Drug Administration for approval of its eteplirsen drug, which will be used to treat Duchenne muscular dystrophy.
A type of muscular dystrophy, DMD causes muscle degeneration. It mainly affects males and can be fatal.
On Monday, April 21, the company announced that it will submit a New Drug Application (NDA) to the FDA by the end of 2014 to get a go-ahead for eteplirsen.
"As we announce our plan to submit an eteplirsen NDA by the end of 2014, we are very pleased with the detailed guidance that the FDA has provided us on a potential eteplirsen approval pathway and their support of a historically controlled eteplirsen confirmatory study," says Chris Garabedian, president and CEO of Sarepta Therapeutics. "We also appreciate that the FDA shares our urgency in dosing a broader base of eteplirsen patients and has encouraged us to begin the clinical program with our follow-on exon-skipping drugs as soon as possible."
This is not the first instance where Sarepta has tried to get an approval for eteplirsen from the FDA. In November 2013, the company's application for eteplirsen was put down by the FDA. The agency said that the data from the company's study of 12 patients was insufficient for an approval. Since then, Sarepta as well as patients' advocates have been trying hard to get FDA to reassess its verdict.
Sarepta says that per the guidelines received from the FDA, it will conduct further studies with eteplirsen. The study will include clinical trials of patients who are between the age of 7 and 16 years and can walk specified distances. The company will also study patients who are younger than 7 years and cannot walk at all.
Sarepta says that it will submit an application for eteplirsen to the regulator under an accelerated approval pathway, which the FDA suggested.
The FDA usually grants accelerated approval to drugs that are used to treat diseases with no treatment options. Sarepta will have to conduct trials on a bigger scale to strengthen its findings. The news undoubtedly breathes new life for kids who are suffering with the fatal disease.
