SRPT Stock Climbs After Sarepta Seeks Permanent FDA Nod For Two Duchenne Therapies

Sarepta is seeking full approval for Amondys 45 and Vyondys 53 in Duchenne muscular dystrophy.

• The FDA accepted the filings for review and set a target decision date of February 28, 2027.
• The applications rely on results from a confirmatory study plus years of real-world data from patients already taking the drugs.
• Sarepta has, however, not filed for full approval of Exondys 51.

Shares of Sarepta Therapeutics (SRPT) rose around 6% on Tuesday after the company announced that U.S. regulators had accepted its applications seeking full, permanent approval for two of its existing medicines for Duchenne muscular dystrophy.

The positive market reaction came after Sarepta revealed that the Food and Drug Administration (FDA) will now review applications for Amondys 45 and Vyondys 53, two treatments already available to certain patients with the rare, progressive muscle-wasting disease.

The FDA accepted the filings for review and set a target decision date of February 28, 2027. These medicines were originally approved on an accelerated, or temporary, basis based on early evidence that they could help. Sarepta is now asking for traditional, full approval, which would give patients and doctors greater long-term certainty about their use in DMD. 

DMD is a serious genetic condition that mainly affects boys and causes muscles to weaken and waste away over time, eventually affecting walking, breathing and heart function.

Key Data Behind The Request

The applications rely on results from a confirmatory study plus years of real-world data from patients already taking the drugs.

The major study called ESSENCE compared the treatments to a placebo. While it did not meet its main statistical goal, it showed consistent positive trends favoring the medicines, including increased production of a muscle-protecting protein and slower loss of function in key daily activities. Further, the treatments were well tolerated over more than two years of use, with no new safety concerns, the company said.

What Comes Next?

The FDA will now conduct a full review of the data. A decision is expected by late February 2027.

If approved, the change to full approval would remove the current requirement for ongoing confirmatory studies and provide stronger regulatory backing for the treatments.

Exondys Pending Full Approval Application

Sarepta has not, however, filed for full approval of Exondys 51, another DMD drug for which it received accelerated approval and belongs to the same category as Amondys 45 and Vyondys 53. Its prescribing information still states that continued approval may depend on confirming clinical benefit in follow-up studies.

The accelerated approval in 2016 for Exondys 51 followed an FDA advisory committee voting against approval and internal reviewers recommending rejection, citing very weak evidence of benefit.

How Did SRPT Retail Traders React?

On Stocktwits, retail sentiment around SRPT stock jumped from ‘neutral’ to ‘bullish’ over the past 24 hours, while message volume remained at ‘high’ levels. According to the platform’s internal data, retail chatter around the stock has risen by about 89% over the past 24 hours.

A Stocktwits user highlighted that the confirmatory trial for Amondys 45 and Vyondys 53 did not meet its primary goal.

Another voiced hopes for a buyout of the company at a significant premium.

According to data from Koyfin, six of the 24 analysts covering SRPT rate it ‘Buy’, while 13 rate it ‘Hold’ and five rate it ‘Sell’ or ‘Strong Sell.’ The 12-month average price target on the stock is $22, representing a potential upside of about 29% from its last closing price.

SRPT stock has fallen 16% year-to-date. 

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