FDA rejects Capricor's cell therapy for Duchenne muscular dystrophy

From my colleague Adam Feuerstein: The Food and Drug Administration rejected a marketing application from Capricor Therapeutics seeking clearance for a cell therapy to treat Duchenne muscular dystrophy, the company said this morning.

In its letter to Capricor, the FDA said the company’s application “does not meet the statutory requirement for substantial evidence of effectiveness” and requested additional clinical data, the company said.

The FDA’s denial of the drug, deramiocel, comes three weeks after the agency’s chief regulator of cell and gene therapies, Nicole Verdun, was removed from her post by her boss, Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research.

As reported exclusively by STAT, Verdun had scheduled an advisory committee meeting to review the Capricor therapy, but Prasad was skeptical of deramiocel and decided unilaterally to cancel the meeting.

REGULATION

FDA publishes rejection letters, but not the most coveted ones

The Food and Drug Administration yesterday published more than 200 complete response letters it sent to drugmakers when it initially rejected their products — but only for drugs that were later approved. The move, while framed as a way to improve transparency and demystify the drug review process, stops short of releasing CRLs for drugs that were never approved, as STAT's Andrew Joseph and Lizzy Lawrence note. And most of the letters released had previously been made public, albeit not all in one place.

“This is the lowest of the low-hanging fruit,” said Peter Lurie, a former associate commissioner for the FDA who has advocated for the release of all CRLs. “It’s the rest of it that’s hard.”

A 2015 analysis found that 85% of drugmakers didn’t mention the FDA’s concerns about safety and efficacy when they announced a rejection. And in 40% of such cases, drugmakers didn’t disclose when the FDA called for a new clinical trial.

stem cells

California's stem cell bank faces sudden demise

The California Institute for Regenerative Medicine is shuttering its once-world-leading iPSC biobank in a fire sale that’s left researchers scrambling and advocates frustrated. Citing low usage and high costs, the agency is offering deep discounts on stem cell lines before a July 16 deadline, STAT’s Jonathan Wosen writes — after which many samples will be destroyed.

Though CIRM will retain archival vials, scientists warn that closing the $32 million taxpayer-funded repository undercuts years of work and hinders future research into complex diseases like Alzheimer’s and autism.

“The biggest challenge and the biggest obstacle for science across the globe has been siloed tools [and] information. CIRM enabled a way to decentralize and make accessible research tools that nobody had,” one of the biobank’s creators told STAT. “It feels we’re going backwards.”

podcast

NIH grant cuts and FDA transparency questions

On this week’s episode of "The Readout LOUD": a closer look at the NIH’s grant-cutting legal playbook, a not-so-transparent transparency push by the FDA commissioner, and another big biotech acquisition.

Our colleague Anil Oza joins us to unravel a previously unseen memo he obtained from HHS that lays out the legal framework being used to justify the termination of NIH grants. The disclosure comes as legal fights over the legitimacy of these funding cuts are being challenged in multiple courts.

We also discuss this week’s announcement that Merck intends to acquire Verona Pharma for $10 billion — another sign that biotech M&A activity is heating up as large pharma companies search for new revenue sources.

Listen here.

vaccines

Moderna's Covid shot for kids gets limited approval

The FDA has granted full approval to Moderna’s Covid vaccine, Spikevax, for children aged 6 months to 11 years — but only for those with underlying medical conditions. The decision aligns with controversial new guidance from health secretary Robert F. Kennedy Jr., who recently revoked vaccine recommendations for healthy children and pregnant people.

“It’s riskier for a very young infant to have any sort of respiratory infection than it is for an older child,” said Fiona Havers, a former CDC epidemiologist who quit last month over Kennedy's vaccination policies. “So this FDA decision is going to leave infants vulnerable to severe disease, and parents who want to protect their baby from severe Covid are going to be left with fewer choices.”

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