Roche Plans New Trial for Muscular Dystrophy Therapy to Seek EU Approval

Dow Jones04-16

By Adria Calatayud

Roche Holding said it would start a late-stage study for a gene therapy for Duchenne muscular dystrophy to seek approval in the European Union after drug regulators in the bloc turned it down last year.

The Swiss drugmaker said Thursday that the new Phase 3 study for the drug, known as Elevidys, aims to generate additional data required for a regulatory resubmission with the European Medicines Agency to make it available to more patients.

The EMA last year recommended the refusal of a marketing authorization for Elevidys, saying the study Roche presented to support its application failed to show the therapy had an effect on movement abilities after 12 months. Roche said at the time it would work with the EMA to explore a path forward.

The company said the new study would test the efficacy and safety of Elevidys compared to placebo over 72 weeks in about 100 boys with Duchenne muscular dystrophy, a rare muscle-wasting genetic disease. The drug seeks to treat the underlying cause of the disease by introducing genetic material with a one-time infusion.

Roche said Elevidys is currently approved in nine countries including the U.S., where Sarepta Therapeutics holds commercialization and manufacturing rights. Last year, Sarepta temporarily paused shipments of Elevidys in the U.S. while drug regulators probed the death of a patient, but resumed them after the investigation concluded that the death was unrelated to the treatment.

Write to Adria Calatayud at adria.calatayud@wsj.com

(END) Dow Jones Newswires

April 16, 2026 08:10 ET (12:10 GMT)

Copyright (c) 2026 Dow Jones & Company, Inc.

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The drug seeks to treat the underlying cause of the dis","collect":0,"end_time":0,"defaultTopTitle":"","property":[],"viewcount":null,"language":"en","relate_stocks":{"BK4007":"制药","03165":"华夏欧优股对冲","RHHBY":"罗氏控股","BK4156":"煤与消费用燃料"},"translate_title":"罗氏计划肌营养不良疗法新试验寻求欧盟批准","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"RHHBY":0.9,"03165":0.6},"content_text":"By Adria Calatayud \n\n\n \n\n\n  Roche Holding said it would start a late-stage study for a gene therapy for Duchenne muscular dystrophy to seek approval in the European Union after drug regulators in the bloc turned it down last year. \n\n\n  The Swiss drugmaker said Thursday that the new Phase 3 study for the drug, known as Elevidys, aims to generate additional data required for a regulatory resubmission with the European Medicines Agency to make it available to more patients. \n\n\n  The EMA last year recommended the refusal of a marketing authorization for Elevidys, saying the study Roche presented to support its application failed to show the therapy had an effect on movement abilities after 12 months. Roche said at the time it would work with the EMA to explore a path forward. \n\n\n  The company said the new study would test the efficacy and safety of Elevidys compared to placebo over 72 weeks in about 100 boys with Duchenne muscular dystrophy, a rare muscle-wasting genetic disease. The drug seeks to treat the underlying cause of the disease by introducing genetic material with a one-time infusion. \n\n\n  Roche said Elevidys is currently approved in nine countries including the U.S., where Sarepta Therapeutics holds commercialization and manufacturing rights. 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