FDA caution is starting to look like cruelty to sick kids

Biomedical research has produced extraordinary breakthroughs that have saved countless lives. But too many promising drugs now stall in federal review, and children with rare diseases are paying the price.

I’m a bioscientist. My work has focused on how healthy cells function and how that knowledge can be applied to therapeutic enzyme development. I’ve spent my career working inside the disciplines that move a treatment from lab bench to patient: protocol design, reproducibility, evidence standards, and layered human testing to ensure safety.

Is this simply bureaucracy doing what bureaucracy does? Or are rare pediatric therapies effectively facing a higher bar inside the system?

Standards, evidence, and process matter. But so does urgency.

Children with rare diseases do not live on regulatory timelines. They lose function month by month — speech, mobility, independence, even the ability to breathe on their own.

Of the more than 6,800 known rare diseases, about 70% begin in childhood. Better-known examples include Duchenne muscular dystrophy, Gaucher disease, and cystic fibrosis.

Developing therapies for these children is difficult, expensive, and slow even under the best conditions. Treatments such as Ultragenyx’s UX111 for Sanfilippo syndrome, Sarepta’s Elevidys for Duchenne, and Regenxbio’s RGX-121 for Hunter syndrome can take decades to develop, years to move through trials, and still more time to reach the children who need them.

That reality makes avoidable regulatory delay even harder to defend.

Too often, applications do not stall because the underlying science has failed. They stall over manufacturing or procedural concerns — in many cases, issues that are fixable and not directly tied to whether the therapy is clinically helping patients. Those delays can undermine the purpose of the FDA’s accelerated approval pathway, which exists to move critical treatments to patients faster while additional data is collected.

As a scientist, I was particularly troubled by the FDA’s recent rejection of a promising Hunter syndrome treatment and by yet another clinical hold placed on its development despite positive trial results.

That raises an uncomfortable question: Does the review process itself need review?

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The approval path for UX111 is another example. The therapy went through the rigorous biologics license application process, only to be delayed by a manufacturing hold.

Elevidys offers a similarly painful lesson. More than 1,200 Duchenne patients received the treatment over three years. Then, after two non-ambulatory patients (including one with underlying complications) tragically died, the FDA pulled the treatment from all patients, leaving families crushed and panicked.

Children are waiting too long for access to potentially life-changing therapies.

Yes, medical breakthroughs have increased. But so have regulatory burdens tied to approval and release. By the time many of these therapies reach the market, a decade or more has passed. In rare pediatric disease, that delay has a name: time children do not have.

Sometimes, it is their entire lifetime.

Manufacturing processes can be improved. Facilities can be upgraded. Paperwork can be corrected.

Lost neurons and muscle fibers cannot be replaced.

FDA leaders, along with Congress and the White House, should push for a smarter accelerated approval process — one that allows multiple requirements to be addressed simultaneously when appropriate, instead of serially dragging out timelines. If regulatory review had moved more efficiently, the Sanfilippo treatment might have cleared on its original 2025 approval timeline. Duchenne patients might not have lost access to the only available gene therapy. Hunter syndrome patients might not still be waiting.

This debate is not about abandoning safety or efficacy standards.

Ultragenyx has said manufacturing improvements are addressable and not directly related to product quality. Sarepeta responded to FDA concerns over Elevidys by requesting black-box warnings while allowing treatment to continue for ambulatory patients. In the RGX-121 Hunter syndrome case, the FDA rejected the use of a long-accepted biomarker (cerebrospinal fluid) used in the trial.

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These decisions do not help children with rare diseases. Timely, science-based approvals would.

And the stakes go beyond today’s patients. Regulatory efficiency also affects whether companies continue investing in rare-disease therapies at all. Orphan drug development requires major upfront investment, long timelines, and often poor financial returns. In many cases, these programs are closer to philanthropic science than blockbuster pharma economics.

When developers face repeated slowdowns across different diseases, sponsors, and technologies for reasons unrelated to core clinical safety or efficacy, the signal to the market is clear: Don’t take the risk.

That is how innovation gets smothered.

At some point, the pattern at the FDA becomes impossible to ignore. Is this simply bureaucracy doing what bureaucracy does? Or are rare pediatric therapies effectively facing a higher bar inside the system?

Those are scientific and ethical questions that deserve honest answers.

Accelerated approval does not mean lower standards. It means applying standards intelligently. It means allowing earlier access while confirming evidence continues to accumulate. It means recognizing that “wait and see” is not neutral. It is a choice that guarantees disease progression in children who cannot afford delay.

Good science and compassion are not competing values. We can maintain rigor and still act with urgency.

The FDA has the authority. The science is moving. The children cannot wait.

Accelerated approval is not cutting corners. It is using every tool we have to save time families do not have.

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