Treatment for Rare Pediatric Cancer Granted Orphan Drug Designation

Cellectar Biosciences announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to CLR 131 for the treatment of rhabdomyosarcoma. 

Rhabdomyosarcoma, a malignant tumor of mesenchymal origin, is the most common type of tissue sarcoma among pediatric patients. CLR 131 is an investigational radioiodinated Phospholipid Drug Conjugate (PDC) drug candidate that delivers cytotoxic radiation directly and selectively to malignant cells. This targeted action reduces radiation exposure to otherwise normal tissues.

CLR 131 is currently being evaluated in a Phase 2 study in relapsed or refractory (R/R) multiple myeloma and various B-cell malignancies (eg, chronic lymphocytic leukemia, small lymphocytic lymphoma, marginal zone lymphoma, lymphoplasmacytic lymphoma, and diffuse large B-cell lymphoma).  The primary endpoint is clinical benefit response (CBR), with secondary endpoints of progression-free survival (PFS), median overall survival (mOS), and other efficacy markers. It is also being evaluated in a Phase 1 study in patients with R/R multiple myeloma exploring fractionated dosing to assess the drug’s safety and tolerability. 

In addition, a Phase 1 study in pediatric solid tumors and lymphoma and another in head and neck cancer (in combination with external beam radiation) are anticipated to begin this year. 

For more information call (608) 441-8120 or visit Cellectar.com.