Sarepta Therapeutics will file an application with the Food and Drug Administration by the end of 2018 seeking accelerated approval for its second drug to treat Duchenne muscular dystrophy, a rare, inherited, muscle-wasting disease.
In an announcement Monday, the company, based in Cambridge, Mass., said the decision to seek a rapid approval of its drug, called golodirsen, was made after receiving a report last week that summarized a meeting with FDA officials held in February.
The meeting, sought by Sarepta to seek regulatory guidance on golodirsen, could have been treacherous. In attendance on the FDA side were clinical reviewers who vehemently disagreed with the agency’s top-level decision in late 2016 to approve Exondys 51, Sarepta’s first drug to treat Duchenne muscular dystrophy (DMD).
My son is 10 yrs old having exon deletion 48-52. currently he is taking deflxacort 18mg 10 days on/off.
Kindly guide me latest treatement/research update status.
You should email the patient advocacy team at Sarepta, Siobhan [email protected] and Allison [email protected], for more information on research and development.
Hi Rajiv our patient centered team is here for you and your son. 1-844-4PTC-CARES. Please also visit our patient website http://www.PTCCares.com
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