March 31, 2025

The need-to-know this morning

AI drug developer Isomorphic Labs raised $600 million.

fda

For biotech, Marks' exit feels like a major blow

Robert F. Kennedy Jr.'s abrupt ousting of Peter Marks — the longtime vaccine and gene therapy czar at the FDA — is being seen as a gut punch for biotech and pharma companies. The industry, once cautiously optimistic about the Trump administration in some respects, is now very much rattled — and time will tell whether it will keep playing nice or finally sound the alarms.

STAT's Adam Feuerstein spent the weekend talking with investors and came away from his conversations thinking biotech, in particular, now has its worst-case scenario realized. For many companies, a crisis in confidence at the FDA is potentially calamitous.

There are concerns that his departure could destabilize the already fragile biotech market, and slow the approval of innovative therapies.

Meanwhile, UC Davis cell biology expert Paul Knoepfler has his own thoughts on the matter.

"Overall, Marks being forced out at CBER highlights what I see as a lack of informed, good intentions behind the so-called Make America Healthy Again movement," he writes opines in a column for STAT. "There is even a potential malevolence behind HHS of late, including its mauling of the NIH. If this continues, it is going to pose many risks to the health of Americans, our country's world-leader role in biomedical science, and our biotech industry."

alzheimer's disease

Europe puts brakes on Lilly's Alzheimer's drug

European Union regulators have advised against the approval of Eli Lilly's Alzheimer's drug Kisunla, citing safety concerns over brain swelling. Though Kisunla showed promise in slowing cognitive decline by up to 35%, the risk of serious side effects — including treatment-related deaths — tipped the scales for the EMA's advisory committee.

The move echoes last year's initial rejection of Biogen and Eisai's Leqembi, which was later overturned on appeal. Now, Lilly faces a similar fork in the road: fight the decision or wait out the bureaucratic purgatory.

radiopharmaceuticals

Pluvicto's expanded FDA nod nearly triples reach

Pluvicto, Novartis' blockbuster radiopharmaceutical for prostate cancer, has been greenlighted by the FDA for use earlier in treatment, nearly tripling the number of patients eligible for its use. Phase 3 data showed a 59% reduction in the risk of disease progression or death, cementing Pluvicto's status as the crown jewel of radioligand therapy.

The treatment brought in $1.4 billion last year, Novartis said in January. The drug might shift treatment protocol and further entrench radiopharmaceuticals as a rising force in oncology.

duchenne muscular dystrophy

Emotional whiplash for PTC Therapeutics

In a twist of regulatory irony, the European Commission has pulled PTC's long-debated Duchenne muscular dystrophy drug Translarna, just as the FDA — which famously rejected it twice — is now giving it a new look. The commission has followed a negative recommendation from the European Medicines Agency, but it's left the door open for individual countries to keep the drug available under special provisions — acknowledging the lack of alternatives for patients with nonsense mutation DMD.

PTC is now shifting to a country-by-country approach with the drug, trying to maintain access where possible in the wake of this disappointing decision.

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