Duchenne Muscular Dystrophy Treatment Makes Little Progress
Hopes are apparently dying that the drug industry will be able to quickly come up with a treatment for an inherited muscle-wasting disorder, known as Duchenne muscular dystrophy, one of the most eagerly researched diseases of recent years.
Progress in understanding the disease triggered a research-and- development bang lately, fueling excitement that pushed the stock prices of some small companies. There are probably 28 trials or studies for potential therapies, which involve a drug for Duchenne or closely linked Becker muscular dystrophy. There are many companies working in the area, including Pfizer Inc. and PTC Therapeutics.
In the middle of intense pressure from the families of the patients, the US Food and Drug Administration (FDA) has decided to accelerate its review of a couple of experimental drugs belonging to BioMarin Pharmaceutical Inc. and Sarepta Therapeutics.
According to BioMarin, the FDA rejected its application to sell its drug, drisapersen, saying that there wasn’t sufficient proof to prove that it was effective.
A briefing document released ahead of an approaching FDA meeting on the drug mentioned that FDA scientists said on Friday that the advantages of Sarepta’s drug seemed inconsistent in a tiny clinical trial, and questioned the research’s methodology.
BioMarin shares have gone nearly 13% down since before the agency’s rejection, and the stock of Sarepta has gone down to about 60% since Friday.
During the FDA’s coming meeting on Sarepta’s drug, eteplirsen, emotions will probably run high when a team of outside experts will suggest the agency on whether it should approve the drug or not. Despite the FDA’s concerns, families and doctors of patients with Duchenne are looking forward to attend the meeting and stand in support for the drug.