August 13, 2020
This week, the U.S. Food and Drug Administration has taken an important step in helping patients with a rare form of muscular dystrophy, giving them hope for a brighter future with a better quality of life.
Duchenne muscular dystrophy (DMD) is a form of the disease affecting mostly boys; almost all are wheelchair-bound by their teens and dead by their 20s. DMD involves enormous patient suffering—loss of ambulation, loss of respiratory function, and loss of ability to complete routine daily functions—and for which there are no cures and only limited treatments that may slow the progression of this cruel disease.
But this week, the FDA granted provisional approval through an accelerated approval process for a treatment, Viltepso (viltolarsen). Viltepso is for patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping, a mutation shared by about 8 percent of DMD patients. Young Jordan McLinn is one of them—and his story is a testament to the importance of giving patients more treatment options and more control over their healthcare decisions.
Jordan was three years old when his family learned that he had DMD. It is impossible for most of us to imagine receiving the news that your precious child had no realistic expectation to recover. But the McLinns still had a glimmer of hope. They learned about a new experimental drug that might help Jordan. Unfortunately, Jordan didn’t qualify for the clinical trial that would allow him to access the drug.
At that same time, Right to Try was an idea being considered in states across the country. Right to Try, a policy designed by the Goldwater Institute that allows those suffering from terminal illnesses to gain access to medication still being tested in clinical trials, while offering legal protection to doctors and drug companies who come to patients’ aid.
In Indiana, young Jordan and his mom became some of the strongest advocates for the policy. They testified before the state legislature, spoke to the media, and met with their then-governor, Mike Pence, who also became a champion for the law.
With the McLinns’ help and tireless advocacy, Right to Try became law in Indiana, was ultimately enacted in 41 states, and was signed into federal law by President Donald Trump in May 2018. The federal law bears Jordan’s name, as well as the names of some of his fellow patient advocates.
In the meantime, Jordan was able to participate in another promising clinical trial—a trial for Viltepso —helping to pave the way for the FDA’s decision this week. Today, Jordan is doing great and isn’t showing any signs of decline. Hopefully, with the FDA’s provisional approval of Viltepso, many more DMD patients can see improvements in their conditions. Jordan’s mom, Laura, wrote on Facebook this week:
“I might never know what it feels like to watch a son score a touchdown at a football game or pin a wrestling opponent on the mat but the way my heart feels now thinking of all he’s sacrificed to make this happen makes my heart just want to explode. I’m so proud of him.”
The Goldwater Institute honored Jordan and Laura for their efforts with the Institute’s first-ever Freedom Award in 2018. Their courage and tireless efforts—while facing the management of Jordan’s illness, as well as business and family obligations—were instrumental in allowing patients to obtain access to investigational treatments.
Right to Try is already saving lives. And thanks to the continued involvement of Jordan in the clinical trial process, some of families that receive the devastating DMD diagnosis now have two new pathways and far more reason to hope than they did just a few short years ago.
“Patients want to access drugs that are FDA-approved,” Laura said in 2018. “If not, they want to access drugs through a clinical trial. If not, they still want to know that there may be some pathway available to them to be able to access the treatments. That’s what Right to Try does. It gives that extra layer of hope.”
This week’s FDA action is an important reminder that thanks to the families like the McLinns, there is much reason to hope.