Cellectar Biosciences (Madison, WI) announced in May that the U.S. Food and Drug Administration (FDA) had granted separate Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation for treatment of neuroblastoma and rhabdomyosarcoma, respectively, with the company’s lead phospholipid drug conjugate (PDC), CLR 131. The approval followed FDA Orphan Drug designation for the treatment for neuroblastoma in March. “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,” said John Friend, MD, chief medical officer of Cellectar. CLR 131 is the first potential therapy that would use a PDC tumor-targeting platform to deliver 131I directly to tumor cells.
In the RPDD for the treatment in neuroblastoma, evidence on targeted molecular radiotherapy of pediatric solid tumors, as originally published in The Journal of Nuclear Medicine (2018;59:244–250), was used as supporting information for the FDA decision. The FDA grants RPDDs for diseases that primarily affect children from birth to 18 years old and affect fewer than 200,000 individuals in the United States. The RPDD may enable Cellectar to receive a priority review voucher that would reduce future FDA review time.
The Orphan Drug Designation for use in rhabdomyosarcoma came only 1 week after the RPDD for use in neuroblastoma.
CLR 131 is an investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the Cellectar’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to tumor cells. CLR 131 is currently in a phase 2 clinical study in relapsed or refractory multiple myeloma and a range of B-cell malignancies, as well as a phase 1 clinical study in patients with relapsed or refractory multiple myeloma, exploring fractionated dosing. In the second half of 2018 the company plans to initiate at the University of Wisconsin Carbone Cancer Center (Madison) a phase 1 study with CLR 131 in pediatric solid tumors and lymphoma. The study will enroll patients with relapsed or refractory pediatric cancers, including 2 cohorts: one with non–brain-related cancers and a second with malignant brain tumors. All enrolled patients will receive a single 30-minute infusion of CLR 131 and will be followed for up to 85 days with safety and efficacy assessments. Cellectar plans another phase 1 study in combination with external-beam radiation for head and neck cancer.
Cellectar Biosciences
- © 2018 by the Society of Nuclear Medicine and Molecular Imaging.