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STAT+: A Rare Disease Drug Was Approvable, Then It Wasn’t. Inside a Surprise Rejection by the FDA

An experimental cell therapy for a rare post‑transplant blood cancer, developed by Atara Biotherapeutics and Pierre Fabre, was initially deemed approvable by FDA reviewers but was abruptly rejected in February 2026 over alleged clinical data deficiencies. The disease affects roughly 500 U.S. patients annually and carries a prognosis of weeks to months. A former FDA employee suggested the reversal stemmed from the agency’s new leadership. The unexpected decision highlights the volatility of regulatory pathways for orphan‑drug candidates.