Niemann-Pick disease, type C (NPC) is a lethal, neurodegenerative disorder that affects children. Presently, no therapies for NPC are approved by the Food and Drug Administration (FDA). Several studies have suggested the potential use of 2-hydroxypropyl-β-cyclodextrin (HPBCD) to treat NPC, but the critical studies and data required for an Investigative New Drug (IND) application to evaluate HPBCD were not available.

On March 10, 2015, the Food and Drug Administration (FDA) approved Unituxin™ (dinutuximab) as part of first-line therapy for pediatric patients with high-risk neuroblastoma, a rare cancer that most often occurs in young children. This approval was the result of a collaborative effort between the National Cancer Institute (NCI), the Children’s Oncology Group, and United Therapeutics Corporation (UTC) for the first approved therapy for pediatric high-risk neuroblastoma.

On March 10, 2015, the U.S. Food and Drug Administration (FDA) approved Unituxin™ (dinutuximab) as part of first-line therapy for pediatric patients with high-risk neuroblastoma, a rare cancer that most often occurs in young children. This approval was the result of a collaborative effort among the National Cancer Institute (NCI), the Children’s Oncology Group, and United Therapeutics Corporation (UTC) for the first approved therapy for pediatric high-risk neuroblastoma.