Sarepta wants to 'own DMD' in rare disease race with BioMarin

Sarepta Interim CEO Edward Kaye

Sarepta ($SRPT), jockeying with rival BioMarin ($BMRN) to commercialize a treatment for Duchenne muscular dystrophy, is looking to buy up complementary assets to fortify its position. But the company's better-funded competitor believes its long history in rare disease R&D will give it a leg up as the pair move toward make-or-break FDA decisions.

Each company is developing a treatment for DMD, a muscle-wasting disease that primarily affects young boys and severely shortens life expectancy. And each has faced setbacks along the way, as critics have questioned whether positive results from tiny studies merit approval. But after months of delays, both treatments will likely be up for FDA decisions over the next 8 months.

In the meantime, Sarepta and BioMarin have escalated their competitive squabble, with each making the case to Bloomberg for its future success in DMD. At Sarepta, interim CEO Edward Kaye said his company is on the hunt for buyout deals that will bring in new muscle-boosting treatments, believing that the company's eteplirsen could eventually star in a combination therapy. "We are going to own DMD," Kaye told Bloomberg, as the company looks to pull the trigger on an acquisition this year and build out its pipeline in the field.

BioMarin, which signed an $840 million deal to acquire Prosensa and get its hands on the DMD-treating drisapersen, is taking a similar approach. Chief Medical Officer Dr. Henry Fuchs told Bloomberg that his company, no stranger to acquisitions, is looking to bulk up in DMD, too. And, unlike Sarepta, BioMarin has the means "that are necessary to actually deliver important medical advances to patients--as opposed to just talking about that," he said.

BioMarin submitted drisapersen in April, and the FDA has promised to make a final decision on the drug by Dec. 27. Sarepta wrapped up its application for eteplirsen last month, setting in motion a 60-day process that precedes a 10-month review. The company is applying for a priority review--which the agency granted to BioMarin--that would shorten that timeframe to 6 months.

Each drug targets the roughly 13% of DMD patients whose disease stems from a flaw in the gene responsible for producing the protein dystrophin. Eteplirsen works by getting the body to skip over that genetic defect, restoring the flow of dystrophin and alleviating symptoms of DMD. The disease affects about 1 in 3,500 newborn boys.

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