FDA Grants Cellectar Rare Pediatric Disease Designation for Experimental Neuroblastoma Treatment

Rare Daily Staff

The U.S. Food and Drug Administration granted Cellectar Biosciences Rare Pediatric Disease designation to CLR 131, the company’s lead therapeutic candidate for the treatment of neuroblastoma, a cancer of the sympathetic nervous system most often found in infants and young children.

CLR 131 is Cellectar’s experimental that targets tumors and selectively delivers radiation to malignant tumor cells while minimizing radiation exposure to normal tissues. CLR 131 is being studied in a number of cancers. The company expects to begin a clinical trial in neuroblastoma in the second half of 2018.

“The grant of RPDD for CLR 131 in conjunction with the orphan drug designation we received in March highlight the critical need for new treatments in the fight against this disease,” said John Friend, chief medical officer of Cellectar.

The FDA grants Rare Pediatric Disease designation for diseases that primarily affect children from birth to 18 years old, and affect fewer than 200,000 persons in the United States. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

If CLR 131 is approved by the FDA for neuroblastoma, the rare pediatric disease designation may enable Cellectar to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive Priority Review for a future NDA or BLA submission which would reduce the FDA review time from twelve months to six months.

The vouchers are potentially lucrative because they are transferable. Most recently, Spark Therapeutics sold a priority review voucher at the end of April for $110 million.

May 2, 2018
Photo: John Friend, chief medical officer of Cellectar