The FDA Just Crushed Sarepta Therapeutics' Hopes Of Releasing A Muscular Dystrophy Drug

Shares of Sarepta Therapeutics Inc SRPT saw a marked decline this week after Food and Drug Administration scientists said studies examining the effects of the firm's latest muscular dystrophy drug were inconsistent.

The FDA's review of Sarepta's drug followed a similar dismissal of BioMarin Pharmaceutical Inc. BMRN's newest muscular dystrophy treatment just a week earlier.

A Blow To Shareholders

Sarepta's results caused the company's shares to fall 63.09 percent over the past week, as many had been expecting the FDA to look more favorably at the firm's treatment after BioMarin's rival therapy drisapersen was rejected. In both cases, the FDA determined that there was not enough research to prove the drugs' benefits, and therefore more studies would need to be carried out.

Related Link: Why BioMarin's Pipeline Could Be Worth $4 Billion Plus

A Difficult Decision

While the FDA's primary objective is to protect patients, many are critical of the agency's quick refusal for both drugs.

Muscular dystrophy claims the lives of its patients, and many families who are struggling with the disease were disappointed to learn that both drugs would be banned from the marketplace until further study. Others say the FDA's strict policies could put pharmaceutical companies off developing muscular dystrophy drugs because it has become too difficult to get one on the market.

A Gold Mine

For companies like Sarepta, Biomarin, Pfizer Inc. PFE and PTC Therapeutics, Inc. PTCT that are working to develop treatments for the disease, a muscular dystrophy therapy that is approved by the FDA would be a gold mine.

Focusing on drugs that treat only a small segment of the population has become a popular strategy among drugmakers, as it allows them to charge thousands of dollars per patient. However, as Biomarin and Sarepta have proven, getting the drug on the shelves can be challenging.

It's Not Over Until It's Over

Both BioMarin and Sarepta are unlikely to give up on their muscular dystrophy treatments. Sarepta is due to have an advisory committee meeting with FDA officials on January 22, where the drug could still be approved despite the agency's most recent comments. Industry experts and muscular dystrophy advocates will be present at the meeting to argue for the drug. However, the chances that the FDA will change its decision now that the documents have been released are slim.