Trump’s FDA Makes the Right Call on Duchenne Therapy

AP Photo/Andrew Harnik, File

He’s been on the job for less than four months, and Dr. Marty Makary’s already having a meaningful impact on patients at the Food and Drug Administration.

This is a new FDA under President Trump — an agency that not only prioritizes patient access and safety but also understands the importance of acting quickly when making a critical decision that impacts patients living with rare and fatal diseases. 

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This week, the Food and Drug Administration lifted the voluntary hold that had been placed on the most promising therapy for a rare and fatal disease affecting young boys. Duchenne muscular dystrophy causes progressive muscle weakness and loss of function. DMD typically appears in early childhood and worsens over time. As the disease progresses, patients are unable to walk or move independently. Eventually, it affects the heart and lungs. 

Today, few Duchenne patients live past their 20s, but advances in research and new treatment options offer families new reasons to hope.

The best hope is a gene therapy, Elevidys, that was originally approved for non-ambulatory patients in June 2023 under the FDA’s accelerated approval pathway. The following year, the gene therapy received traditional approval for ambulatory patients. 

Like so many other new treatments, recent cases raised questions about whether the gene therapy is effective for patients in the latter stages of Duchenne disease progression when they become non-ambulatory. In response, the FDA did what it always does. It launched an investigation, pressured the company to voluntarily remove the drug from the market, and evaluated the efficacy for all patients taking the treatment. 

The FDA made the right call to pull the therapy for non-ambulatory patients based on recent cases. It should restrict access to a population of patients for whom it manifestly did not work. 

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Patients also know that usually this caution is taken too far — to a place of decision paralysis.  A few days ago, a senior official at the FDA was projecting an “arduous and treacherous path” for the gene therapies’ approval. For these boys, time is not a luxury; it is a rapidly dwindling resource. A prolonged period of FDA indecision would have been a direct assault on the very hope that these young patients cling to.  

This time, the FDA acted differently. It was cautious, deliberate — and for the first time — swift.  

Because these boys don’t have a lot of time. 

In a matter of days, the FDA conducted a thorough investigation and concluded that the gene therapy should remain an option for ambulatory patients living with Duchenne.

A treatment or therapy that works for one population but not another is not a failure – it’s a lifesaving cure. So many times, we have advocated for therapies and medications that we thought would save a life or could save a life. This is different: We know this therapy is helping some boys in the earlier stages of Duchenne. Patients who are still able to walk are showing progress with increased mobility. 

Rare disease patients need the FDA’s default position to be access to the latest innovation — if there’s evidence that such treatments can help or heal them. Patients living with rare and fatal diseases know better than anyone else the risks associated with their treatments. The choice should be theirs — not the choice of a government committee.

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During his first term, President Trump signed into law the “Right to Try Act,” a landmark legislation that allows terminally ill patients to access experimental treatments when other approved treatment options have failed. The President promised then to put patients back in charge of their health decisions.  

He’s doing it again. 

The FDA’s Duchenne decision ensured the public’s safety, demonstrated its interest in the patient perspective, and managed to be quick on its feet. We should all celebrate the recent actions by President Trump’s FDA — with Marty Makary in charge.

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