Second Death in Gene Therapy Trial for a Rare Muscle Degenerative Disorder

'Regulatory flexibility' issues surrounding the case could affect development of other gene therapies, including those financed by California taxpayers.

Sarepta graphic

A second death has occurred involving a gene therapy for Duchenne muscular dystrophy, and the company involved has halted shipments of the treatment.

Sarepta Therapeutics announced the death in a news release at 1 a.m. EDT on Sunday. The publicly traded firm said it was also pausing an ongoing phase three clinical trial of the therapy, Elevidys, that targets older ambulatory and non-ambulatory patients.

Duchenne is a rare genetic disorder mainly affecting boys. It is characterized by progressive muscle degeneration. The average life span is 18 to 25 years.

“We are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patient’s family and his care team during this incredibly difficult time,” Louise Rodino-Klapac, Sarepta’s chief scientific officer, said in a statement.

The first death was of a 16-year-old boy in March. The company did not release the age or sex of the person in the most recent case.

The Sunday announcement received limited attention but it is likely to be the source of more commentary today. The issues involved also resonate with the taxpayer-supported California Institute for Regenerative Medicine (CIRM), which has backed 116 stem cell and gene therapy clinical trials.

CIRM has funded five research projects dealing with Duchenne, according to its website.

“The death is certain to add additional scrutiny to the FDA’s decision last year