Cellectar Biosciences (Nasdaq:CLRB), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, today announces that the U.S. Food and Drug Administration(FDA) Office of Orphan Products Development has granted Orphan Drug Designation to CLR 131, the company’s lead Phospholipid Drug Conjugate™ (PDC) product candidate, for the treatment of neuroblastoma, a rare pediatric cancer.
“Neuroblastoma is the third most common childhood cancer for which there are currently no approved treatments for children with relapsed or refractory disease,” stated John Friend, M.D., chief medical officer of Cellectar. “The FDA’s granting of orphan drug designation for CLR 131 highlights the significant need for new treatments for children with neuroblastoma, and we believe that the targeted delivery of CLR 131 represents a promising novel approach to its treatment.”
Orphan drug designation provides seven year market exclusivity benefit, increased engagement and assistance from the FDA, tax credits for certain research, research grants and a waiver of the New Drug Application user fee. Neuroblastoma is recognized by the FDA as an orphan disease, usually defined as a condition that affects fewer than 200,000 people nationwide.
The FDA previously accepted the Company’s Investigational New Drug application for a Phase 1 open-label, dose-escalating study to evaluate the safety and tolerability of a single intravenous administration of CLR 131 in up to 30 children and adolescents with cancers including neuroblastoma, sarcomas, lymphomas (including Hodgkin’s lymphoma) and malignant brain tumors. Cellectar expects to initiate this study during the second quarter of 2018. Read the full release here.