|Sarepta Therapeutics Inc. NDA Submission to FDA for Eteplirsen for treating Duchenne Muscular Dystrophy|
|By Josh Gee|
|Monday, 29 June 2015 19:34|
Developer of RNA-targeted therapeutics Sarepta Therapeutics Inc. (NASDAQ:SRPT) has confirmed completion of rolling submission of New Drug Application to United States Food and Drug Administration for eteplirsen on June 26, 2015.
The firm’s main drug Eteplirsen is looking to cure Duchenne muscular dystrophy and is made for enabling production of functional internally shortened in dystrophin protein in patients having mutations.
Edward M. Kaye, interim chief executive officer and chief medical officer, opined that there are over 13% of people having Duchenne muscular dystrophy and estimated to have mutation.
Kaye added completion of NDA submission for eteplirsen demonstrates finishing employee efforts, sites as well as patients of Duchenne community.
The firm is looking forward in working with FDA during regulatory process for chasing goal of bringing eteplirsen to patients suitable to exon 51 skipping whereas maintaining organizational focus on moving PMO technology for targeting other DMD subpopulations amenable to exon-skipping as quickly as possible.
The NDA submission has request for Priority Review. Earlier, eteplirsen has been approved Orphan and Fast Track status by FDA.
The rolling submission of NDA started on May 20, 2015 after finishing a pre-NDA meeting with FDA held on May 19, 2015.
Shares of SRPT fell as much as 8.60% to $28.58 on higher than average volume in Monday’s session.
Sarepta Therapeutics, Inc. is a biopharmaceutical company. The Company is focused on the discovery and development of unique ribonucleic acid (RNA-targeted therapeutics for the treatment of rare, infectious and other diseases. The Company is primarily focused on advancing the development of its potentially disease-modifying Duchenne Muscular Dystrophy (DMD) drug candidates, including its lead DMD product candidate, eteplirsen, which is an antisense PMO therapeutic in Phase III clinical development for the treatment of individuals with DMD who have an error in the gene coding for dystrophin that is amenable to skipping exon 51.