FDA, Sarepta and gene therapy
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Roche Holding AG paused some shipments of the controversial gene therapy Elevidys following a similar action by the embattled US biotechnology company that developed the treatment.
Roche is following its partner Sarepta and halting shipments of the Duchenne gene therapy Elevidys in some countries, amid safety concerns.
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Sarepta Therapeutics laying off dozens in Ohio, won't comply with FDA request to halt therapy
After the FDA request, Cambridge, Massachusetts-based Sarepta said in a statement that it will continue to ship the therapy to ambulatory people but maintain a halt it implemented June 15 for non-ambulatory patients after reporting to the FDA a case of acute liver failure in a patient who could not walk.
The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational gene therapy trials for limb girdle muscular dystrophy following three patient deaths potentially linked to the company's treatments.
A 51-year-old man died last month after receiving an experimental treatment from Sarepta, the third death this year tied to the company's gene therapies
The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated with Duchenne MD.
Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta Therapeutics adds a black box warning to the gene therapy for acute liver injury and failure and parts with more than a third of employees.