STAT+: A rare disease drug was approvable, then it wasn’t. Inside a surprise rejection by the FDA

An experimental therapy for a rare blood cancer was on the path toward approval by the Food and Drug Administration last year — with internal reviewers recommending it be cleared — before the agency rejected the drug last month, according to people familiar with the matter.

The cell therapy, being developed by Atara Biotherapeutics and Pierre Fabre Pharmaceuticals, is intended to treat a type of cancer that can occur following a stem cell or 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.

The FDA rejection, on the grounds that clinical data on the drug were deficient, was a “complete reversal that I can’t help but think was due to the FDA’s new leadership,” a former agency employee with direct knowledge of the review, speaking on the condition of anonymity, told STAT.

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