18:46 , Feb 15, 2019 |  BC Week In Review  |  Clinical News

Sarepta's golodirsen under FDA Priority Review

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said Feb. 14 that FDA accepted and granted Priority Review to its NDA for golodirsen (SRP-4053) to treat Duchenne muscular dystrophy amenable to skipping exon 53. its PDUFA date is Aug....
16:45 , Feb 14, 2019 |  BC Extra  |  Company News

Priority Review for Sarepta's golodirsen to treat DMD

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said FDA accepted and granted Priority Review to its NDA for golodirsen (SRP-4053) to treat Duchenne muscular dystrophy amenable to skipping exon 53. its PDUFA date is Aug. 19. Sarepta is...
17:09 , Mar 16, 2018 |  BC Week In Review  |  Clinical News

Sarepta to submit NDA for DMD candidate by YE

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said it plans to complete submission of a rolling NDA to FDA by year end for golodirsen (SRP-4053) to treat Duchenne muscular dystrophy amenable to exon 53 skipping. The company said...
18:39 , Mar 14, 2018 |  BC Extra  |  Clinical News

Sarepta resumes dosing at U.K. sites after DMD trial halt

Sarepta Therapeutics Inc. (NASDAQ:SRPT) President and CEO Doug Ingram said the company has resumed dosing at U.K. sites in the Phase III ESSENCE trial evaluating golodirsen (SRP-4053) and casimersen (SRP-4045) to treat Duchenne muscular dystrophy...
21:47 , Mar 12, 2018 |  BC Extra  |  Company News

Sarepta to complete NDA submission for DMD candidate by YE18

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said on Monday that it plans to complete submission of a rolling NDA to FDA by year end for golodirsen (SRP-4053) to treat Duchenne muscular dystrophy amenable to exon 53 skipping....
20:59 , Feb 16, 2018 |  BC Week In Review  |  Clinical News

U.K. trial hold for Sarepta's SRP-4045 combo

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said it halted dosing at U.K. study sites in the Phase III ESSENCE trial evaluating golodirsen (SRP-4053) and casimersen (SRP-4045) to treat Duchenne muscular dystrophy. In a statement, the company said...
20:58 , Feb 16, 2018 |  BC Week In Review  |  Clinical News

U.K. trial hold for Sarepta's SRP-4053 combo

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said it halted dosing at U.K. study sites in the Phase III ESSENCE trial evaluating golodirsen (SRP-4053) and casimersen (SRP-4045) to treat Duchenne muscular dystrophy. In a statement, the company said...
22:20 , Feb 9, 2018 |  BC Extra  |  Clinical News

Sarepta's DMD trial halted in U.K. on safety signal

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said it halted dosing at U.K. study sites in the Phase III ESSENCE trial evaluating golodirsen (SRP-4053) and casimersen (SRP-4045) to treat Duchenne muscular dystrophy. Sarepta shares were off $2.03 to...
19:11 , Oct 13, 2017 |  BioCentury  |  Regulation

Seeking surrogates

Thanks to flaws in PTC Therapeutics Inc. ’s trials, the recent FDA advisory committee meeting to discuss Translarna ataluren did nothing to clarify what level of dystrophin expression would be necessary to support an accelerated...
19:47 , Sep 8, 2017 |  BC Week In Review  |  Clinical News

Sarepta reports Phase I/II data for exon 53 DMD candidate

Sarepta Therapeutics Inc. (NASDAQ:SRPT) reported data from 25 boys with Duchenne muscular dystrophy (DMD) with confirmed deletions of the DMD gene amenable to skipping exon 53 from the Phase I/II 4053-101 trial showing that once-weekly...