Unituxin Approval Leads to Rare Priority Review Voucher Award
United Therapeutics announced that the Food and Drug Administration (FDA) has granted a Rare Pediatric Priority Review Voucher (PPRV) as part of the Biologics License Application (BLA) approval for Unituxin (dinutuximab), for neuroblastoma.
Unituxin is a disialoganglioside, GD2-binding chimeric monoclonal antibody indicated, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2), and 13-cis-retinoic acid (RA), for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy. The safety and effectiveness of Unituxin was evaluated in a randomized, open-label, multicenter trial conducted in pediatric patients with high-risk neuroblastoma. All patients had received prior therapy consisting of induction combination chemotherapy, maximum feasible surgical resection, myeloablative consolidation chemotherapy followed by autologous stem cell transplant, and radiation therapy to residual soft tissue disease.
The PPRV was created in 2012 by the Food and Drug Administration Safety and Innovation Act (FDASIA) to encourage development of treatments for rare pediatric diseases. To qualify to receive a PPRV, a sponsor must submit an application for a drug or biologic intended to prevent or treat a rare pediatric disease. The rare pediatric disease application also must be eligible for priority review and rely on clinical data derived from studies examining a pediatric population and dosages of the drug intended for that population. The voucher can be sold and the holder of the voucher can redeem it with a subsequently filed New Drug Application or BLA, requiring FDA to meet the review goals for a priority review, as opposed to a standard review.
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