STAT+: A rare disease drug was approvable, then it wasn\u2019t. Inside a surprise rejection by the FDA
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An experimental therapy for a rare blood cancer was on the path toward approval by the Food and Drug Administration last year \u2014 with internal reviewers recommending it be cleared \u2014 before the agency rejected the drug last month, according to people familiar with the matter.<\/p>\n
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 \u2014 children and adults \u2014 and leaves them with weeks or months to live.<\/p>\n
The FDA rejection, on the grounds that clinical data on the drug were deficient, was a \u201ccomplete reversal that I can\u2019t help but think was due to the FDA\u2019s new leadership,\u201d a former agency employee with direct knowledge of the review, speaking on the condition of anonymity, told STAT.<\/p>\n
Continue to STAT+ to read the full story\u2026<\/a><\/p>\n<\/div>\n