2:47 PM
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. The company said it will seek accelerated approval for golodirsen based on an increase in dystrophin protein as a surrogate endpoint. The announcement follows a February meeting with FDA's Division of Neurology Products.

In 2016, FDA approved Sarepta's Exondys 51 eteplirsen to treat DMD amenable to exon 51 skipping. The agency based its approval of Exondys 51 on the same surrogate endpoint of an increase in dystrophin. Exondys 51 is a phosphorodiamidate morpholino (PMO) that induces skipping of exon 51 dystrophin mRNA (see BioCentury, Oct. 13, 2017).

In a Phase I/II trial in 25 boys with DMD, golodirsen increased mean dystrophin protein levels to 1.019% of normal at week 48 compared to 0.095% of normal at baseline as measured by Western blot (p<0.001) (see BioCentury Extra, Sept. 6, 2017).

Sarepta is evaluating golodirsen and casimersen (SRP-4045) in the Phase III ESSENCE trial to treat DMD amenable to exon 53 or 45 skipping, respectively. According to the company, FDA said ESSENCE may serve as a postmarketing confirmatory study for golodirsen if the candidate receives accelerated approval.

Golodirsen is a PMO targeting exon 53 and casimersen is a PMO targeting exon 45.

Sarepta gained $4.74 to $79.10 on Monday.

User Sign in

Trial Subscription

Get a 4-week free trial subscription to BioCentury Extra

Article Purchase

Purchase this article for limited one-time distribution and website posting

$750 USD