Duchenne Gene Therapy Faces Crisis: Roche Halts Elevidys for Vulnerable Patients After Fatalities

• Roche has immediately halted its gene therapy, Elevidys™, for all non-ambulatory Duchenne muscular dystrophy (DMD) patients globally.
• This drastic measure follows two tragic patient deaths from acute liver failure, a known risk now under urgent review^{1, 2, 7}.
• Crucially, treatment for ambulatory DMD patients is not affected, with their benefit-risk profile remaining positive^{2, 3, 5}.

A groundbreaking gene therapy, once a beacon of hope for Duchenne muscular dystrophy, now faces a critical safety crisis. Roche announced the immediate suspension of Elevidys™ (delandistrogene moxeparvovec) for all non-ambulatory patients, both in commercial use and clinical trials, following the heartbreaking news of two patient deaths due to acute liver failure^{1, 2, 7}.

The devastating fatalities, occurring among approximately 140 non-ambulatory individuals treated globally with the AAV-vector gene therapy^{5, 7}, have cast a somber shadow over Elevidys. This has prompted an urgent reassessment by Roche, concluding an "unfavourable" benefit-risk profile for those DMD patients who have lost mobility. "We are deeply saddened by the loss of these two young men," stated Levi Garraway, M.D., Ph.D., Roche's Chief Medical Officer. "Patient safety is always our highest priority"¹.

Effective immediately, commercial dosing for non-ambulatory patients is stopped. Clinical trial enrollment for this group, including the ENVISION study, is paused worldwide until additional risk mitigation measures, potentially including enhanced immune modulation, are implemented^{1, 3, 5}. This decision sends ripples through the gene therapy landscape, underscoring the significant, though rare, risks tied to AAV-based treatments⁵.

For ambulatory DMD patients, however, the path forward with Elevidys remains unchanged; treatment continues as its benefits are still deemed to outweigh the risks in this population^{2, 3, 5}. Roche and its partner Sarepta are now in urgent collaboration with health authorities and independent expert panels, racing to implement enhanced safety protocols as the Duchenne community watches with bated breath^{2, 3, 6}.

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References

1. www.clinicaltrialsarena.com
2. www.chugai-pharm.co.jp
3. www.parentprojectmd.org
4. defeatduchenne.ca
5. www.ainvest.com
6. www.roche.com
7. www.parentprojectmd.org
8. medically.roche.com