America deserves an FDA that drives progress. But under Commissioner Makary, the agency remains the nation’s bottleneck—watching promising treatments gather dust, even as RFK Jr.’s Make America Healthy Again (MAHA) movement expands access to food reform and state-level health initiatives.
In July, the FDA issued Complete Response Letters (CRLs) to Replimune’s RP1 melanoma therapy and Capricor’s Duchenne cardiomyopathy treatment—not for safety, but for trial design technicalities, despite breakthrough designations from the agency itself. These rejections come at great cost: gene therapies that once sparked hope are held back by regulatory caution, not reason. Most recently, the FDA rejected Ultragenyx’s Sanfilippo gene therapy over manufacturing concerns—again, not patient safety.
Then Elevidys, the sole approved gene therapy for Duchenne muscular dystrophy, faced an FDA shutdown. After three patients died from complications, FDA demanded a voluntary halt, only to reverse course days later amid backlash from patient groups and Senator Ron Johnson. If deaths signal safety risk, why not compassionate access first for terminal patients with no alternatives?
These delays aren’t minor—they are failures of leadership. Right-to-Try laws exist precisely to serve patients like those denied RP1 or Capricor treatments. Yet under Makary, access pathways remain crippled by regulatory opacity. No clarity on what manufacturers must do to qualify. No pre-approved hurdles clarified for desperate families. Promises of transparency in Complete Response Letters remain unfulfilled.
This is not speculation. It’s systematic. Makary’s FDA emphasizes procedural symmetry over therapeutic urgency—rewarding form over function. Actions speak louder than tweets: while RFK Jr. takes food dye off the national menu, Makary’s team stands motionless, waiting.
The message to biotech innovators is chilling: expect delays, celebrate rejections, and abandon hope if you aim to move fast. When Gill decision is dictated by rigid rule interpretation rather than patient need—when a deadly disease meets a regulatory quagmire—America loses.
Time is running out: With Trump’s administration winding down, MAHA’s legacy hinges on results, not rhetoric. Admin offices bargain with Big Food for healthier labels, but the nation’s rarest disease patients await results only promised in bureaucratic briefs.
Congress must act: hold hearings, subpoena FDA officials, and demand to know why groundbreaking treatments are denied for cosmetic reasons. FDA leadership must grant certitude: what level of evidence fulfills Right-to-Try? What trial designs is FDA willing to negotiate on? If sponsors act in good faith, FDA must facilitate—not frustrate—access.
The alternative is tragic: patients losing parents, counties losing hope, and the U.S. forfeiting its claim as a biotech powerhouse. Makary’s FDA, once glossed as innovation-friendly, now feels like a lockbox on progress.
Commissioner Makary still has time to course-correct. He must leave behind the days of "No" and bring an FDA that says "Yes—when science and need align." He can rewrite the agency’s reputation by freeing therapies that patients deserve and critics fear. But only if actions align with promise.
America taught the world gene science. Now we must teach it delivery. Under current leadership, regulatory caution becomes moral failure. There’s no margin left for delay—not when patients draw their last breaths waiting.
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