Investors Gear Up For Exciting News as FDA Sets Dates for BioMarin and Sarepta DMD Drugs

Published: Jul 21, 2015

Investors Gear Up For Exciting News as FDA Sets Dates for BioMarin and Sarepta DMD Drugs
July 21, 2015
By Mark Terry, Breaking News Staff

The U.S. Food and Drug Administration (FDA) set its calendar yesterday for two big decisions regarding drugs from BioMarin Pharmaceuticals and Sarepta Therapeutics for Duchenne muscular dystrophy.

Cambridge, Mass.-based Sarepta is competing directly with San Rafael, Calif.-based BioMarin for a drug to treat Duchenne muscular dystrophy (DMD). On June 29, 2015, Sarepta announced it had completed the rolling submission of a New Drug Application (NDA) for eteplirsen for DMD.

BioMarin beat Sarepta to the FDA with its own NDA in April for drisapersen. Both drugs utilize “exon skipping” technology, which allows the damaged cells’ repair mechanisms to fix the specific genetic mutation that affects about 13 percent of individuals with the disease.

The FDA’s 2015 Advisory Committee Tentative Meetings calendar was updated yesterday and the Peripheral and Central Nervous System Drugs Advisory Committee, which would look at BioMarin’s drisapersen and Sarepta’s eteplirsen is tentatively scheduled for Nov. 23 and 24.

BioMarin’s drisapersen filing has an approval decision date of Dec. 27. Sarepta filed for eteplirsen on June 29, but is still waiting for formal acceptance and an approval date.

Adam Feuerstein, writing for TheStreet, refers to the Nov. 23 and 24 dates as “an epic event for biotech investors.”

DMD primarily affects boys and its onset is typically between the ages of three and five. Survival rates have improved recently, but the most common age of mortality is in the early 30s, though there are cases of men surviving into their 40s and 50s. DMD results in progressive muscle degeneration and weakness.

Edward Kaye, chief executive of Sarepta, recently said the company was looking to buy a drug to treat DMD. The reasoning behind this would be to potentially mix the new drug with eteplirsen for a cocktail of treatments.

“We are going to own DMD,” Kaye said in an interview with BloombergBusiness, indicating the company wanted “a menu of options.”

The rivalry between the two companies is fairly well known and Henry Fuchs, chief medical officer of BioMarin, said in an interview with BloombergBusiness, “With the Duchenne muscular dystrophy space you betcha we’re going to be creating and owning franchise spaces.” And furthermore, he said that BioMarin has the resources “that are necessary to actually deliver important medical advances to patients — as opposed to just talking about that.”

Fighting words, to be sure. But BioMarin has about $1.01 billion in cash to go shopping with, while Sarepta has $166 million. On the other hand, Sarepta has only $6.4 million in debt compared to BioMarin’s $656 million.

The two companies sparred at a San Francisco investor meeting in January, when BioMarin’s Jean-Jacques Bienaime, the company’s chief executive officer, publically said that its drug data for DMD was superior to Sarepta’s. Chris Garabedian, then chief executive officer of Sarepta, criticized Bienaime for inappropriate comments and then went on to attack the way BioMarin analyzed its drug data.

For investors, Nov. 23 and 24 are likely to be interesting dates for both insights into the two company’s drug development plans, but for the drama of a potential catfight.

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