| After Sarepta’s dramatic back-and-forth with the Food and Drug Administration, I started reaching out to patients and their families to see how they were feeling about the company’s controversial gene therapies. On Monday I called Christine McSherry, a Massachusetts mother whose adult son has taken an older Sarepta drug to treat Duchenne muscular dystrophy. Two teenage boys died of acute liver failure in recent months after taking Elevidys, a Sarepta drug, to treat the muscle-wasting disease. And McSherry said she had heard about numerous cases of boys who ended up hospitalized with liver problems linked to the Sarepta therapy, mostly from social media groups. Even though her son hasn’t taken Elevidys, she said she is still alarmed by the lack of information from the drugmaker about the recent safety controversy. “I got worried about the silence that was around these deaths,” said McSherry, a founder of Jett Foundation, a patient group named after her son. “All these families are looking for answers and no one is giving answers.” Last month, after talking to several affected families, she filed a citizen petition demanding that the FDA do something about Elevidys, including adding a boxed warning about the potential for liver toxicity. She wants the Cambridge, Massachusetts-based company to release more information on Elevidys and better communicate with families of patients taking or considering the drug. “There is no reasonable — or legal — basis for omitting death and the potential for hospitalization from the Elevidys labeling,” she wrote in the petition.  Sarepta headquarters in Cambridge, Massachusetts. Source: Sarepta Therapeutics Inc. FDA leaders met with Sarepta, the agency said in a statement Friday, and requested it voluntarily stop all shipments of the drug, which is its biggest product. “The company refused to do so,” the FDA said. After facing backlash for that decision, Sarepta reversed course on Monday and announced that it planned to temporarily pause all Elevidys shipments in the US. The pause, which will start at the end of the day Tuesday, will allow the company to respond to information requests from the FDA, Sarepta said in a statement. Elevidys is a key drug for Sarepta, making up more than half of the company’s product sales in its most recent quarter. Sarepta’s troubles intensified last week when the company said that a 51-year-old patient died of acute liver failure in June in an early-stage trial of a gene therapy to treat limb-girdle muscular dystrophy. The FDA on Friday said it placed a hold on gene therapy clinical trials for that specific muscle disease. In light of the controversies, McSherry said Sarepta needs to be more forthcoming with its customers. “They keep saying over and over again, we are so transparent,” she said. “If they are so transparent, why do we keep seeing all the adverse events on social media and not hearing it from them?” — Bob Langreth | 
