Edition:
United Kingdom

Sarepta Therapeutics Receives Complete Response Letter From The U.S. Food And Drug Administration For Golodirsen New Drug Application


Monday, 19 Aug 2019 

Aug 19 (Reuters) - Sarepta Therapeutics Inc ::SAREPTA THERAPEUTICS RECEIVES COMPLETE RESPONSE LETTER FROM THE US FOOD AND DRUG ADMINISTRATION FOR GOLODIRSEN NEW DRUG APPLICATION.SAREPTA THERAPEUTICS INC - CRL GENERALLY CITES TWO CONCERNS.SAREPTA THERAPEUTICS INC - SAREPTA WILL IMMEDIATELY REQUEST A MEETING WITH FDA TO DETERMINE NEXT STEPS..SAREPTA THERAPEUTICS INC - CRL CITES CONCERNS ABOUT RISK OF INFECTIONS RELATED TO INTRAVENOUS INFUSION PORTS AND RENAL TOXICITY.SAREPTA - RENAL TOXICITY WITH GOLODIRSEN WAS OBSERVED IN PRE-CLINICAL MODELS AT DOSES THAT WERE TEN-FOLD HIGHER THAN DOSE USED IN CLINICAL STUDIES.SAREPTA THERAPEUTICS INC - RENAL TOXICITY WAS NOT OBSERVED IN STUDY 4053-101, ON WHICH APPLICATION FOR GOLODIRSEN WAS BASED..SAREPTA - OVER COURSE OF REVIEW, AGENCY DID NOT RAISE ANY ISSUES SUGGESTING NON-APPROVABILITY OF GOLODIRSEN, INCLUDING ISSUES THAT FORMED BASIS OF CRL.SAREPTA - GOT CRL FOR NDA SEEKING ACCELERATED APPROVAL OF GOLODIRSEN INJECTION FOR DMD TREATMENT IN PATIENTS WITH CONFIRMED MUTATION AMENABLE TO EXON 53 SKIPPING.