Sarepta reports 3rd patient death after gene therapy treatment

A third patient has died from acute liver failure after receiving a gene therapy treatment from Sarepta Therapeutics, the biotech company confirmed to Bloomberg on July 18. 

The patient died while receiving Elevidys, a gene therapy treatment approved to treat Duchenne muscular dystrophy, a rare genetic disorder that progressively weakens skeletal and heart muscles. Two other patient deaths linked to acute liver failure were reported in March and June. The latest death involved a patient with limb-girdle muscular dystrophy, a disease that weakens muscles in the hips and shoulders, who was enrolled in an early stage clinical trial.

In a statement shared with Bloomberg, the company said it notified investors and regulators about the latest death “in an appropriate and timely manner.” 

“While we do everything possible to ensure patient safety, there is inherent risk in clinical trials, and we are grateful to the courageous patients and families who participate,” the statement said. 
In a “strategic restructuring and pipeline prioritization” plan released July 16, Sarepta said it will add a black box warning for liver failure to the Elevidys label at the request of the FDA. The company said the FDA’s concerns about using Elevidys in patients who can walk, or ambulant patients, were resolved through the addition of the warning label. However, Sarepta is still working with regulators on new safety protocols for non-ambulant patients.

Sarepta also said July 16 it will cut 36% of its workforce and pause several drug programs to save $400 million annually.