Marty McCurry Resigns from FDA Leadership

In light of all the troubling news out there, let’s take a moment to acknowledge something that feels a bit more positive: Marty McCurry has resigned from his position as head of the Food and Drug Administration. He was just sworn in last week, yet his time there was anything but smooth.

McCurry’s brief tenure was overshadowed by various issues such as internal scandal, forced resignations, and a notable decline in staff morale. Some would argue that his approach even hindered President Trump’s efforts to expand clinical trials for rare diseases through the “Right to Try” framework, which aims to give patients more access to experimental treatments that aren’t fully approved yet.

Essentially, the philosophy behind Right to Try is pretty basic. Patients with rare conditions—and their families—often feel like they have nothing to lose when facing dire circumstances. The hope is that governments won’t be the barrier preventing them from innovative solutions that could save lives.

However, McCurry’s leadership seemed to be at odds with these ideals. For instance, there were numerous requests from the MPS community to meet with him, but they were met with generic, automated responses. When Senator Ron Johnson highlighted these concerns, the FDA claimed approval rates had “peaked,” even as they struggled to approve necessary treatments, like drugs aimed at conditions linked to mucopolysaccharidosis.

This bait-and-switch truly frustrated many families, leading to dramatic protests—one being a mock funeral outside the FDA’s offices by dozens of mothers advocating for better treatment options for their children. MPS disorders can lead to serious health problems, including various physical and developmental issues, yet the FDA has delayed approvals for many promising treatments.

Mark Dant from the Ryan Foundation emphasized that the agency’s consistent disapproval of certain accelerated pathways means the lives of children hang in the balance. As he poignantly stated, families know the treatments are there, yet they can’t access them.

While McCurry’s exit might not fix everything, it certainly presents an opportunity for new leadership to act. The next appointees should aim to revive the original spirit behind the Right to Try initiative—prioritizing swift access to effective treatments for children.

Around the globe, parents have long taken to the streets for their children’s well-being. For instance, in Georgia, some have protested outside government buildings for over 500 days, striving to provide their kids with the best chances at life. It’s disheartening to think that American families might need to resort to similar tactics to be heard.

The new FDA leadership should clarify their stance on clinical trial rights, facilitating better communication with families about what would be required for drug approvals. Transparency must be prioritized, as children’s lives aren’t just points in a bureaucratic debate; they’re real stakes in a fight against serious illness.

If McCurry’s resignation helps to open up this dialogue, there might finally be a glimmer of hope for families battling ultra-rare diseases.