On May 2, 2018 Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation (RPDD) to the company’s lead phospholipid drug conjugate, CLR 131, for the treatment of neuroblastoma (Press release, Cellectar Biosciences, MAY 2, 2018, View Source [SID1234525957]).

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"Neuroblastoma is a devastating cancer most often found in infants and young children. 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, M.D., chief medical officer of Cellectar. "We look forward to working with the FDA to bring this potential therapy to pediatric patients and expect to begin a clinical study in neuroblastoma during the second half of 2018."

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 U.S. 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. Currently, these vouchers can also be transferred or sold to another entity. Over the last 16 months, five priority review vouchers were sold for between $110 million to $150 million each.

About Neuroblastoma

Neuroblastoma, a neoplasm of the sympathetic nervous system, is the most common extracranial solid tumor of childhood, accounting for approximately 7.8% of childhood cancers in the United States and is recognized by the FDA as an orphan disease. The incidence is about 10.54 cases per 1 million per year in children younger than 15 years and 90% are younger than 5 years at diagnosis. Approximately 50% of patients present with metastatic disease requiring systemic treatment. Although the prognosis is favorable in children under one year of age with an 86% to 95% 5-year survival, in children aged one to 14 years the 5-year survival ranges from 34% to 68%.

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131, is in a Phase 2 clinical study in relapsed or refractory (R/R) MM and a range of B-cell malignancies and a Phase 1 clinical study in patients with (R/R) MM exploring fractionated dosing. In the second half of 2018 the company plans to initiate a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma, as well as a Phase 1 study in combination with external beam radiation for head and neck cancer.