Sarepta Liver Failure: Setback & Future Outlook
- (NASDAQ:) experienced a significant setback after a second patient death linked to its ELEVIDYS treatment for Duchenne muscular dystrophy (DMD).
- The company has halted ELEVIDYS shipments for non-ambulatory patients and retracted its revenue guidance.
- Investors are eyeing Solid Biosciences (NASDAQ:), a small-cap biotech firm developing STG-003, a perhaps safer alternative to ELEVIDYS.
Sarepta Therapeutics faced a important blow as a second patient death linked to its ELEVIDYS treatment for Duchenne muscular dystrophy triggered acute liver failure (ALF). The company immediately halted shipments to non-ambulatory patients and retracted its financial guidance, leading to a significant stock plunge. This setback has cast a shadow over the gene therapy market, with investors now closely monitoring Solid Biosciences and its potential alternative, STG-003. The impact of this advancement, highlighted by News Directory 3, underscores the inherent risks within medical advancements. While Sarepta aims to mitigate ALF risks, the future of ELEVIDYS and its market share is uncertain. Discover what’s next …
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Sarepta Stock Plummets After ELEVIDYS Death; Solid Bio Gains
Updated June 19, 2025
Sarepta Therapeutics Inc. (NASDAQ:) experienced a significant setback after a second patient death linked to its ELEVIDYS treatment for Duchenne muscular dystrophy (DMD). the patient died from acute liver failure (ALF) following treatment, mirroring a similar incident three months prior. This news sent Sarepta’s stock reeling.
The company has halted ELEVIDYS shipments for non-ambulatory patients and retracted its revenue guidance. Wall Street analysts responded with a wave of downgrades June 16, causing Sarepta’s stock to plunge more than 42%.
The DMD gene therapy market now faces uncertainty. Investors are eyeing Solid Biosciences (NASDAQ:), a small-cap biotech firm developing STG-003, a perhaps safer alternative to ELEVIDYS. Solid Biosciences’ shares jumped more than 11% the same day, signaling confidence in their gene therapy candidate.
ELEVIDYS previously accounted for 61% of Sarepta’s $612 million in revenue last quarter and fueled most of its growth,with sales surging 180%. The company had projected 2025 sales of $2.3 billion to $2.6 billion, implying a 29% increase over 2024. These projections are now in question.
Sarepta is limiting the ELEVIDYS shipment suspension to non-ambulatory patients, who represent a smaller portion of their patient base. The company estimates that it can remain profitable by continuing sales to ambulatory patients. Sarepta is also developing an enhanced treatment regimen to mitigate the risk of ALF, pending FDA approval.
Analysts tracking Sarepta maintain a consensus price target of around $70, suggesting a potential upside of more than 230%. This reflects the company’s ability to continue selling ELEVIDYS to a majority of patients. However, another patient death could further depress Sarepta’s shares. The FDA review of sarepta’s enhanced regimen could take considerable time.
Solid Biosciences is positioning STG-003 as a viable alternative to ELEVIDYS. Early trial data suggests high efficacy. In February, the company reported STG-003 achieved the highest average microdystrophin expression of any DMD gene therapies to date, a key indicator of treatment effectiveness.
While ELEVIDYS has been associated with elevated liver enzymes in about 30% of patients, STG-003 appears to be less damaging to the liver. Solid Biosciences presented data at the jefferies Global Healthcare Conference on June 4, noting that