FAIRLESS HILLS, PA (AP) – BioMarin Pharmaceutical shares plunged by a third after U.S. regulators rejected its potentially game-changing gene therapy for hemophilia A patients.
The Food and Drug Administration late Tuesday raised concerns Roctavian might not really be a one-and-done lifetime treatment, so it wants study participants to be followed longer.
BioMarin now will have to complete an ongoing late-stage patient study, delaying possible approval till late 2022.
The therapy could have freed hemophilia A patients from frequent, extremely expensive IV infusions of blood-clotting therapy to prevent internal bleeding from the rare genetic disorder.
Questions about how long Roctavian works arose recently amid suggestions the company could charge up to $3 million per patient.
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