Sarepta Planning to Dominate Duchennes in Rare Disease Race With BioMarin

July 17, 2015

By Damian Garde


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.


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