FDA to reconsider treatment for rare cancer after its surprise rejection

Two companies developing a therapy for a rare blood cancer have reached an agreement with the Food and Drug Administration that walked back the agency’s main reason for rejecting the drug in January, STAT's Adam Feuerstein reports.

Pierre Fabre Pharmaceuticals and Atara Biotherapeutics, makers of the drug called Ebvallo, said this morning that a meeting held in late April with FDA officials ended with the agency agreeing that their already completed, single-arm clinical trial was sufficient to support a review and potential approval.

When the FDA rejected Ebvallo, the agency said the same study was flawed and the data produced from it was “insufficient” to support the drug’s approval. That decision shocked and frustrated Pierre Fabre and Atara because they said at no point during the review cycle did the FDA inform them that there were concerns or deficiencies with the Ebvallo study or data, the companies told Adam at the time.

The companies have more work to do before resubmitting Ebvallo to the FDA.

drug pricing

Trump report projects massive savings from secret drug pricing deals

The Trump administration is claiming its “most-favored nation” drug-pricing framework could save the U.S. $529 billion over a decade, STAT’s Daniel Payne writes. It plans to tie the cost of new drugs with the second-lowest net price in peer countries like Canada, Japan, and Germany. The analysis, written by the White House Council of Economic Advisers, also projected another $64 billion in Medicaid savings — though the underlying deals with drugmakers remain confidential.

The report revealed companies would self-report international net prices using government guidance designed to avoid violating foreign confidentiality laws, while prices would also be adjusted for U.S. purchasing power. But the details are still murky.

“There’s just no way to verify it by the information that’s public so far,” said Rachel Sachs, a Washington University professor of law who focuses on health care. “It’s been difficult because we don’t know exactly what manufacturers are agreeing to.”

fda

FDA leadership clash stalls Sanofi diabetes drug review

Sanofi has asked the FDA to remove its type 1 diabetes drug teplizumab from Commissioner Marty Makary’s new rapid-review voucher program. The decision stems from an internal dispute at the agency in which acting drug center chief Tracy Beth Høeg opposed the staff’s plans to approve the treatment, sources familiar with the matter told STAT’s Lizzy Lawrence.

The agency has already missed its April decision deadline, and Høeg’s intervention has fueled broader concerns that Makary’s high-profile voucher initiative is opening the door to political interference in scientific drug reviews. Career staff believed the risks tied to teplizumab — Høeg was concerned about Epstein-Barr virus reactivation and possible cancer risk — could be managed through labeling, and the FDA’s surveillance office has not linked the drug to cancer. But Høeg reportedly remained unconvinced and moved toward convening an advisory committee.

“I'd be incredibly disappointed if politics got in the way of science here,” said Aaron Kowalski, CEO of patient advocacy group Breakthrough T1D.

M&A

Angelini buys Catalyst Pharma and its rare disease drugs for $4.1B

The Italian company Angelini Pharma said this morning that it would buy rare-disease focused Catalyst Pharmaceuticals for roughly $4.1 billion in cash, STAT's Andrew Joseph writes.

The deal values Florida-based Catalyst at $31.50 a share, a 28% premium to the 30-day period before April 22, when it became publicly known that a deal was in the works.

Catalyst's top-selling drug Firdapse is approved to treat Lambert-Eaton myasthenic syndrome, known as LEMS, in patients age 6 and older. The drug was at the center of a closely watched fight involving Catalyst and another company also developing a LEMS therapy and the Food and Drug Administration. The decade-long skirmish involved Catalyst suing the FDA at one point and the company drawing criticism for the price tag it set for the drug.

Biotech

Next-gen Duchenne drug from Entrada disappoints

From my colleague Jason Mast: Entrada Therapeutics’ next-generation drug for Duchenne muscular dystrophy disappointed in an early trial, raising questions about the company’s competitiveness in an increasingly crowded field.

Entrada is one of a group of companies developing new exon-skipping drugs. These medicines are designed to help patients with certain mutations produce shortened but still functional forms of dystrophin, the protein missing in Duchenne.

cancer

Color bets AI can scale modern cancer care

Color Health is betting that the future of oncology is primarily online, STAT’s Angus Chen writes. CEO Othman Laraki argues that the exploding complexity of cancer along with workforce shortages and rising costs make traditional models increasingly unsustainable.

“In our mind, the only way this is going to be addressed and solved is in a virtual first, AI-driven manner,” Laraki said. “In the coming years, the biggest cancer centers will be virtual first.”

Color Health has now expanded into a virtual cancer clinic that uses AI tools, multidisciplinary tumor boards, and partnerships with outside experts to help guide diagnosis and treatment decisions — all while coordinating with local oncologists rather than replacing them. The model just received certification from the American Society of Clinical Oncology, giving it a rare external validation in a field where virtual care remains relatively untested.

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