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news.The US Food and Drug Administration (FDA) has granted rare pediatric disease designation for Sarepta Therapeutics' lead drug candidate eteplirsen, a potential treatment for patients with duchenne muscular dystrophy (DMD) who are amenable to skipping exon 51.
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Eteplirsen is designed to address the underlying cause of DMD by allowing the production of a functional dystrophin protein.
The company said that data from clinical trials of eteplirsen in DMD patients have showed a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression.
This rare pediatric disease designation supplements the FDA’s orphan drug and fast track Status previously granted for eteplirsen.
Sarepta interim chief executive officer and chief medical officer Edward Kaye said: "We are pleased that the FDA’s Office of Orphan Products Development has granted eteplirsen with a Rare Pediatric Disease Designation.
"We appreciate that FDA has created the Rare Pediatric Disease Priority Review Voucher program to foster development of treatments for rare pediatric diseases, a core focus for the Company, and we hope it leads to expedited treatments for children who desperately need them."
Sarepta is a biopharmaceutical firm focused on the discovery and development of new RNA-targeted therapeutics for the treatment of rare, infectious and other diseases.
Mainly, the company is focused on advancing the development of its potentially disease-modifying DMD drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51.