| Back in 2018, Jordan McLinn, a young boy with a rare muscle disorder, was photographed hugging Donald Trump as the president signed the Right to Try law aimed at making it easier for dying patients to access experimental drugs. Last month, Jordan’s mother, Laura McLinn, and Senator Ron Johnson, who had sponsored the law, played a behind-the-scenes role in helping families whose children have Duchenne muscular dystrophy regain access to a gene therapy after sales were paused following patient deaths due to acute liver failure. As we reported this week, they were among the parents and politicians who mobilized to pressure the Food and Drug Administration to reverse its stance and allow the use of Sarepta Therapeutics’s Elevidys to resume. A key part of the Right to Try law is to allow terminally ill patients who have run out of options to seek access to medicines that haven’t yet been approved.
Sarepta says on its website that it doesn’t give its experimental drugs through the right to try pathway, saying that could jeopardize the company’s ability to conduct research studies.
Sarepta’s Elevidys received a broad approval by the FDA in 2024. Its approval was marred by controversy. Agency reviewers recommended against it being given, citing insufficient data showing it works. But a top FDA official at the time overruled their objections, clearing it for the vast majority of children with Duchenne, including those who could no longer walk.
After deaths were linked to the drug, the FDA, under new leadership, asked Sarepta to pause shipments in July.
Johnson said he views Elevidys as a “right to try issue” and that regulators “way too often become risk averse.” He said he spoke with FDA Commissioner Marty Makary about how to get the drug back on the market. The FDA says it “is continuing to investigate the risk of acute liver failure” for gene therapies that use Sarepta’s technology. Over the past seven years, few patients have used the Right to Try law. But Johnson’s involvement in advocating for Sarepta’s drug was a reminder that the spirit of the law hasn’t gone away. In fact, it may have more support in Trump’s second term. Health Secretary Robert F. Kennedy Jr. said recently that if people want to take unapproved therapies, they ought to be able to do that, even if there are risks. He said he went to Antigua to get a stem cell treatment for his throat, which is affected by a neurological disorder called spasmodic dysphonia. Parents of children with Duchenne are desperate for anything that might help curb the muscle-wasting disorder in their loved ones, many of whom are in a wheelchair by their teens and won’t live past their 30s. To some experts, the Sarepta story raises questions over how much power patient groups and right to try advocates should have over the FDA. The agency’s job is to weigh the safety and efficacy of a drug before approving it. While patients should have a voice at the agency, it’s unclear what the limit ought to be, said Holly Fernandez Lynch, an associate professor of medical ethics at University of Pennsylvania.
“If all the FDA does is say ‘patients are willing to try it,’ then we face the question of why do we have the FDA at all?” she asked. — Gerry Smith and Robert Langreth | 
