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FDA Sets Review Dates for Muscular Dystrophy Drugs

Analysts expect sparks to fly between competing companies

The FDA has marked its calendar for much-anticipated reviews of two new drugs for Duchenne muscular dystrophy (DMD) –– drisapersen and eteplirsen. BioMarin Pharmaceutical, Inc., the developer of drisapersen, and Sarepta Therapeutics, the developer of eteplirsen, are in a head-to-head race to be the first to market a new treatment for DMD.

BioMarin submitted a new drug application (NDA) for drisapersen in April 2015, and Sarepta followed with an NDA for eteplirsen in June. Both drugs use “exon skipping” technology to skip exon 51 of the dystrophin gene.

Drisapersen is an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of DMD amenable to single exon skipping. The drug induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to DMD patients for whom skipping of exon 51 restores the proper dystrophin reading frame, corresponding to approximately 13% of DMD patients.

Eteplirsen uses phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene. By skipping exon 51, eteplirsen may restore the gene’s ability to make a shorter, but still functional, form of dystrophin from messenger RNA. Promoting the synthesis of a truncated dystrophin protein is intended to stabilize or significantly slow the disease process in patients with DMD.

The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has tentatively scheduled review dates of November 23 and November 24 for drisapersen and eteplirsen, respectively. The former drug has already received an approval-decision date of December 27.

Sarepta recently said it was looking to buy a drug to treat DMD. Observers believe that the reasoning behind this would be to potentially mix the new medication with eteplirsen for a cocktail of treatments.

DMD primarily affects boys, and its onset is typically between the ages of 3 and 5 years. Survival rates have improved recently, but the most common age of mortality is in the early 30s, although there are cases of men surviving into their 40s and 50s. DMD results in progressive muscle degeneration and weakness.

Sources: BioSpace; July 21, 2015; BioMarin Pharmaceutical; June 29, 2015; and Sarepta Therapeutics; April 21, 2014.


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