European Medicines Agency Requests Pause on Trials for Sarepta’s DMD Gene Therapy
Sarepta and Roche have temporarily halted three European clinical trials of their DMD gene therapy Elevidys, following an EMA request after a 16-year-old patient death in the U.S. The paused trials, including studies in boys and newborns, are on hold pending analysis of the death’s cause. Four U.S.-based Elevidys studies continue.
Analysts believe the EMA’s pause will likely be temporary. The patient’s death was attributed to acute liver failure, a known Elevidys risk. Sarepta acknowledged a possible Elevidys contribution.
The death has increased safety concerns about AAV gene therapies, like Elevidys, which delivers microdystrophin. Sarepta noted the patient’s recent viral infection as a possible factor.
Sarepta’s share price dropped 42%. Analysts noted the patient’s age likely meant a higher dose, but maintained a 60% EU approval projection.
Elevidys’ U.S. approvals faced controversy due to trials not meeting endpoints and its $3.2 million price. Despite this, sales are strong, with projected annual sales of $2 billion.
https://www.fiercepharma.com/pharma/after-patient-death-sarepta-and-roche-pause-3-trials-gene-therapy-elevidys-europe
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