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news.US-based biopharmaceutical firm Sarepta Therapeutics has held a pre-new drug application (NDA) meeting with the US Food and Drug Administration (FDA) regarding its lead product candidate, eteplirsen, to treat duchenne muscular dystrophy (DMD).
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After completing the meeting with the agency, the company has agreed to initiate a rolling NDA submission and will submit the non-clinical and CMC components of the NDA by the end of this week.
The company is planning to submit the final component of the NDA by mid this year.
Sarepta interim CEO and chief medical officer Edward Kaye said: "The initiation of our NDA submission for eteplirsen marks a significant milestone for the Duchenne community and we look forward to completing the submission by the middle of the year and to continuing to work with the Agency towards the goal of providing treatments to patients as quickly as possible."
Sarepta is focused on the discovery and development of new RNA-targeted therapeutics to treat 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.
The company is also developing therapeutics to treat drug-resistant bacteria and infectious, rare and other human diseases.