When President Trump and HHS Secretary RFK Jr. rolled out Make America Healthy Again (“MAHA”), it sounded like a rebuke to Big Government and Big Pharma ambitions alike. But today, the FDA — ostensibly Mr. Kennedy’s regulatory anchor — has quietly become the firmest wall between gene-therapy breakthroughs and needy Americans.
Marty Makary’s leadership has shown us what happens when big ambitions outrun institutional capacity or courage.
Consider Replimune’s RP1 therapy: a potential advance in melanoma treatment that earned FDA breakthrough designation and showed a nearly 33% overall response rate in patients who had already failed PD-1 inhibitors.
Yet on July 22, the agency issued a Complete Response Letter — not over safety concerns, but citing the phase 1/2 IGNYTE trial as “not adequate” or well-controlled, and complaining about heterogeneous patient populations.
In other words, science-based innovation was excoriated for statistical imperfections.
Then there’s Capricor’s deramiocel cell therapy for Duchenne-induced cardiomyopathy.
Drafted for priority review and expected to be approved, the FDA slammed it anyway with a CRL, citing gaps in manufacturing data and effectiveness evidence — despite prior internal guidance that the therapy was on track.
Capricor executives have said they addressed nearly all those concerns — with clearly communicated materials — but the agency issued rejection just weeks before the PDUFA date, without reviewing them.
The only concession?
Employers must resubmit and restart the clock.
Patients with DMD?
Still waiting.
Most troubling of all is what happened with Sarepta’s Elevidys, the first FDA-approved gene therapy for Duchenne muscular dystrophy.
After deaths in older patients, Makary’s agency asked for a voluntary pause on shipments.
Sarepta initially refused, citing lack of evidence the drug caused harm. Within days, after fierce backlash — including action by Sen. Ron Johnson, R-Wis., — FDA reversed course and allowed the therapy to resume for ambulatory kids.
But the reversal lacked transparency, and the pause cut off access for families in crisis, especially those already enrolled under Right-to-Try.
Speaking of Right-to-Try, advocacy has collapsed into policy noise.
The law was championed as a lifeline — only to see patients and physicians encountering vague company rules and opaque FDA definitions.
Key leaders like Johnson and Trump praised it; Makary’s FDA has offered policy ambiguity instead. Pressed in congressional hearings, the agency cannot say what qualifies under the law — and no evidence shows any streamlined access mechanism.
The gap between legislative intent and regulatory execution remains wide.
Meanwhile, FDA staffing meltdown signals broader dysfunction.
Just this week, Coya Therapeutics disclosed that the agency missed its IND review deadline for a Phase 2 ALS trial — not because the data don’t reach threshold, but because FDA lacked staff to handle its workload.
Coya was told decisions may stretch into August. When the nation’s most vulnerable remain tied to FDA’s staffing chaos, medicine dies of delay
Finally, personnel drama at the top: Dr. Vinay Prasad, assigned by Makary to lead CBER, lasted only three months. He was pilloried by activists.
Conservatives called for him to be fired; conservatives prevailed.
Though Makary describes him as “a genius,” Prasad is now gone amid speculation of ideological misalignment.
The fact that Makary still speaks publicly of consulting with Prasad suggests regulatory incoherence at the conceptual level.
Patients needing breakthrough therapies for terminal illnesses, gene therapies on life’s edge, or early ALS treatment are not looking for negotiation — they are seeking coherence and access.
And every bureaucratic blunder, every staff delay, and every leadership flub diminishes legitimacy.
President Trump still controls FDA funding and leadership restructuring.
He should consider this agency his healthcare legacy battleground.
If Makary can’t slay the delays and align leadership with MAHA’s urgency, then Washington must consider leadership that will.
Patients are waiting — innovation shouldn’t.
Mitchell Brown is an Army veteran, with extensive experience as a linguist, intelligence collector, and reconnaissance asset. Mr. Brown eventually took a legislative role in the U.S. House, during which he began his work on policy for the chairman of the Committee on Homeland Security. Mitchell was subsequently appointed to serve as deputy White House Liaison for the Department of Labor for the Trump administration. He was also tasked with leading in lowering the unemployment rate during COVID-19. Read Mitch Brown’s Reports — More Here.
© 2025 Newsmax. All rights reserved.
