FDA added a boxed warning to Elevidys after two pediatric deaths and imposed stricter monitoring rules.
- FDA added a boxed warning to Elevidys after two pediatric deaths and imposed stricter monitoring rules.
- Use of gene therapy is now limited to walking DMD patients aged four and older.
- The update deepens a tough stretch for Sarepta following a failed trial and declining Elevidys revenue.
Sarepta Therapeutics shares fell over 1% in premarket trading on Monday after the U.S. Food and Drug Administration approved new labeling for the company’s gene therapy Elevidys, adding its most serious safety warning and sharply narrowing the group of Duchenne muscular dystrophy (DMD) patients eligible to receive the drug.
FDA Tightens Restrictions Following Two Deaths
The agency added a boxed warning to Elevidys after two non-ambulatory pediatric patients died from acute liver failure following treatment. The revised label removes authorization for all non-ambulatory patients, limiting use to walking patients aged four and older.
In July, Sarepta had voluntarily paused distribution of the $3.2 million therapy for non-ambulatory patients after regulators issued a safety alert. The new labeling now formalizes that restriction and further shrinks the potential market for the treatment.
Parent Project Muscular Dystrophy, a major advocacy group, said the update “serves as a reminder that while gene therapy breakthroughs bring promise and hope, serious questions remain about the safety and long-term outcomes of these therapies,” adding that the changes give families “a clearer understanding of who is eligible … and what monitoring is required,” according to a Reuters report.
Expanded Monitoring And New Post-Market Study Required
Under the revised label, patients must undergo weekly liver function tests for at least three months, and remain close to an appropriate medical facility for two months after treatment.
The FDA is also requiring Sarepta to run an observational study involving about 200 DMD patients, tracking liver function and safety outcomes for at least 12 months after receiving Elevidys.
Setback Caps A Difficult Year For Sarepta
The label revision follows a string of setbacks for the company. Earlier this month, Sarepta said its Phase 3 ESSENCE trial for two other DMD drugs, Amondys 45 and Vyondys 53, failed to meet its main goal, showing no statistically significant benefit on stair-climb speed after 96 weeks.
The company attributed part of the disappointing outcome to trial disruptions during the pandemic, stating that results looked more favorable when data affected by the pandemic was excluded.
Financial pressures have also intensified. In the third quarter, revenue declined to $399.4 million from $467.2 million a year earlier, in part due to reduced Elevidys sales following June’s suspension for non-ambulatory patients. Sarepta posted a $179.9 million net loss, reversing last year’s profit, while cash reserves dropped to $865 million from $1.5 billion.
Stocktwits Bulls See Room For A Run
On Stocktwits, retail sentiment for Sarepta was ‘neutral’ amid ‘low’ message volume.
One user said, “$25 is a reasonable target for this week. Next week we can start talking 30+.”
Another user said, “lets have a great week! Hopefully the biotech sector will continue to enjoy a good influx of money, acquisitions continue, and more good news for SRPT!“
Sarepta’s stock has declined 85% so far in 2025.
For updates and corrections, email newsroom[at]stocktwits[dot]com.<