- With additional data from its confirmatory late-stage trial, Sarepta is now seeking to shift the approval type to traditional for the two DMD therapies.
- Oppenheimer analyst Andreas Argyrides sees the sNDA filing as positive as it removes the risk of Amondys/Vyondys being pulled from the market for the next year.
- Earlier this week, Sarepta also said that screening and enrollment are underway in a cohort of its study aimed at developing an enhanced safety protocol for its DMD gene therapy Elevidys.
Sarepta Therapeutics (SRPT) said on Thursday that it intends to make certain submissions to the U.S. Food and Drug Administration regarding two of its Duchenne muscular dystrophy therapies by the end of April, in a move applauded by Wall Street.
Sarepta is now looking to submit supplemental new drug applications (sNDA) seeking conversion of the accelerated approvals of Amondys 45 and Vyondys 53 to traditional approvals, with the help of data from a confirmatory late-stage study.
The company said that the FDA has confirmed that data from the trial can be submitted as part of the application. However, the adequacy of the data to support conversion to traditional approval will be a matter of review, the company said.
Analyst Takeaway
Oppenheimer analyst Andreas Argyrides sees the sNDA filing as positive as it removes the risk of Amondys/Vyondys being pulled from the market for the next year. The analyst added that it also signals positive FDA feedback during a meeting, pointing to potential sNDA approval.
The firm reiterated an ‘Outperform’ rating on Sarepta with a price target of $37 on the shares.
About Vyondys And Amondys
Vyondys 53 and Amondys 45 are both FDA-approved, weekly intravenous antisense oligonucleotide therapies from Sarepta for Duchenne muscular dystrophy (DMD), a severe, progressive genetic disorder primarily affecting young boys, characterized by muscle degeneration. Both therapies now have an accelerated approval.
While Accelerated Approval (AA) allows faster market access for drugs treating serious conditions, based on surrogate endpoints that likely predict clinical benefit, Traditional Approval requires direct evidence of clinical benefit. Accelerated approval requires post-marketing confirmatory trials to prove clinical efficacy to prevent the approval from being withdrawn.
With additional data from its confirmatory late-stage trial, Sarepta is now seeking to shift the approval type to traditional for the two DMD therapies.
Sarepta’s Update For Elevidys
Earlier this week, Sarepta also said that screening and enrollment are underway in a cohort of its study aimed at developing an enhanced safety protocol for its gene therapy, Elevidys, in non-ambulant individuals with Duchenne muscular dystrophy.
FDA-approved Elevidys came under scrutiny last year over concerns of acute liver failure and associated patient deaths. While the company resumed shipments to ambulatory patients after a temporary pause in July 2025, it is now working to address the safety concerns in patients who cannot walk.
How Did Stocktwits Users React?
On Stocktwits, retail sentiment around SRPT stock stayed within the ‘bearish’ territory over the past 24 hours, while message volume remained at ‘low’ levels.
SRPT stock is down 79% over the past 12 months.
For updates and corrections, email newsroom[at]stocktwits[dot]com.