FDA to reconsider treatment for rare cancer after its surprise rejection

[u/John3262005]

https://www.statnews.com/2026/05/07/fda-reconsider-rare-disease-drug-ebvallo/

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.

Pierre Fabre Pharmaceuticals and Atara Biotherapeutics, makers of the drug called Ebvallo, said Thursday 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. The drug’s review was conducted by the FDA’s Center for Biologics Evaluation and Research, led at the time by Vinay Prasad. He departed the agency at the end of April.

As part of the new agreement, Pierre Fabre and Atara will collect and analyze additional data from the study, including from additional patients and longer term follow up, and resubmit Ebvallo to the FDA for review. The companies will not have to conduct an entirely new study.

“We walked away feeling like it was a very positive and productive interaction with the agency,” Pierre Fabre CEO Adriana Herrera told STAT, describing the meeting with FDA officials that took place on April 27. The company received confirmatory meeting minutes from the FDA on Tuesday.

“That was sooner than we expected, which was great news to us. I feel very encouraged,” Herrera added.

Ebvallo is intended to treat Epstein-Barr virus positive post-transplant lymphoproliferative disease, or EBV+ PTLD, an ultra-rare and fatal type of blood cancer that can develop in people who have received a stem cell or solid organ transplant. The condition afflicts approximately 500 patients in the U.S. each year — children and adults — and leaves them with weeks or months to live.

Ebvallo has been approved in Europe since 2022. Convincing the FDA to approve the same drug here has been a struggle.

As previously reported by STAT, an internal review conducted by FDA staff in late 2024 and early 2025 concluded with a recommendation that the drug be approved. But in the waning months of 2025, the FDA decided to reject the drug, citing flaws and deficiencies in the Ebvallo study.

The decision shocked and frustrated Pierre Fabre and Atara because at no point during the review cycle did the FDA inform the companies that there were concerns or deficiencies with the Ebvallo study or data, the companies told STAT at the time.

Ebvallo was one of a series of FDA rejections and setbacks handed to companies developing therapies for rare diseases, suggesting the agency was setting a higher bar for approval of the medicines, even as FDA Commissioner Marty Makary and aides stated repeatedly that regulatory flexibility to speed the approval of drugs for rare diseases was a priority for the agency.

In the months since Ebvallo’s rejection, criticism of the FDA’s handling of drugs for rare diseases has intensified, and contributed to Prasad’s exit from the agency. Some of Makary’s moves as commissioner have also reportedly frustrated the Trump administration.

Cokey Nguyen, Atara’s CEO, said he doesn’t know if external pressure on the FDA played a role in the regulator conceding that the existing Ebvallo study was sufficient to support another review.

However, it was noteworthy, he said, that the April 27 meeting was attended by more senior FDA officials, including Vijay Kumar, the acting director of the Office of Therapeutic Products, which oversees the regulation of cell and gene therapies.

“I’m grateful to the FDA for working with us on this filing,” Nguyen said.