United Therapeutics Corp. (UTHR) announced Tuesday that the U.S. Food and Drug Administration has approved its Unituxin (dinutuximab) injection to treat a deadly form of pediatric cancer.
Unituxin, in combination with granulocyte-macrophage colony-stimulating factor, interleukin-2, and 13-cis-retinoic acid, has been approved for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy.
Unituxin is only the third drug that has received initial FDA approval for a pediatric cancer in over 20 years.
"This approval has been a collaborative effort between the National Cancer Institute (NCI), the Children's Oncology Group and United Therapeutics for the first approved therapy for pediatric high-risk neuroblastoma. We are grateful for the FDA's thorough review and collaboration on this program, and we look forward to expanding our research efforts in the area of pediatric oncology, United Therapeutics' President and Co-CEO.
Neuroblastoma is a type of cancer that most often occurs in young children. It is the most common extracranial solid cancer in childhood and the most common cancer in infancy, with an annual incidence in the U.S. of about 700 patients, of whom 50 percent are diagnosed as having high-risk disease. Unituxin is an antibody that binds to the surface of neuroblastoma cells.
The approval was based on demonstration of improved event-free survival (EFS) and overall survival (OS) in a multicenter, open-label, randomized trial (ANBL0032) conducted by the Children's Oncology Group (COG).
The safety and efficacy of Unituxin were evaluated in a clinical trial of 226 pediatric participants with high-risk neuroblastoma whose tumors shrunk or disappeared after treatment with multiple-drug chemotherapy and surgery followed by additional intensive chemotherapy and who subsequently received bone marrow transplantation support and radiation therapy.
FDA, as part of the Biologics License Application approval for Unituxin, awarded United Therapeutics a Rare Pediatric Priority Review Voucher for neuroblastoma, a rare pediatric disease.
The voucher can be sold, and the holder of the voucher can redeem it with a subsequently filed New Drug Application or Biologics License Application, requiring FDA to meet the review goals for a priority review, as opposed to a standard review.
United Therapeutic's Unituxin is the second pediatric priority review voucher and the first pediatric cancer voucher to be awarded.
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