Cellectar Biosciences Inc.'s lead drug candidate CLR 131 received the U.S. Food and Drug Administration's rare pediatric disease designation.
The designation is meant for the treatment of rhabdomyosarcoma, a rare cancer that affects children. The FDA previously granted orphan drug status to the treatment in the same indication.
The drug also previously received the rare pediatric disease designation by the U.S. regulator as a treatment for neuroblastoma — a cancer that forms in nerve tissue and primarily affects infants and young children.
The designation is granted to medicines that primarily affect children from birth to 18 years old and fewer than 200,000 persons in the U.S. It is intended to encourage the development of new drugs and biologics to prevent and treat rare pediatric diseases.
If CLR 131 is approved by the FDA for either neuroblastoma or rhabdomyosarcoma, the rare pediatric disease designation may enable Cellectar to receive a priority review voucher.
The voucher can be used to reduce the FDA review time by half to six months and can be transferred or sold to another entity. Over the last 16 months, five priority review vouchers were sold for between $110 million and $150 million each, the company noted.
