Sarepta Therapeutics (NASDAQ:SRPT) will no doubt be thanking Adam Feuerstein, writer for thestreet.com, after his article entitled: “Here's How Sarepta Can Ultimately Win FDA Drug Approval” caused shares to surge 20% in pre-market trading.
His report suggested the biotech firm could still secure approval of its muscular dystrophy treatment despite a negative panel vote by the US Food and Drug Administration.
On Monday, members of the advisory panel decided that clinical data for its eteplirsen drug to treat Duchenne muscular dystrophy wasn't strong enough to justify approval.
Eteplirsen uses a novel technology which partially corrects the genetic defect, allowing muscle cells to produce a somewhat functional form of dystrophin, the protein otherwise lacking in sufferers of the disease.
“The negative outcome was a devastating disappointment to the boys stricken with DMD and their families attending the panel,” wrote Feuerstein.
The FDA has until May 26 to make a decision, but ultimately the decision relies with senior FDA official Dr Janet Woodcock.
Ahead of the panel decision, Woodcock, the agency’s top drug evaluator, spent a considerable amount of time making supportive statements, said Feuerstein, she also spent time talking with several affected families.
As Director of the Center for Drug Evaluation and Research she has the power to overrule the decision made by the panel.
It is estimated that around 10,000 young boys suffer from the muscle wasting disease in the US.
Boys with DMD are often wheelchair-bound by their teens, with a life expectancy of at most their late twenties.