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FDA grants orphan drug designation to CLR 131 for Ewing sarcoma
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The FDA granted orphan drug designation to CLR 131 for treatment of Ewing sarcoma, according to the agent’s manufacturer.
CLR 131 (Cellectar Biosciences) is an investigational radioiodinated phospholipid drug conjugate therapy designed to exploit the tumor-targeting properties of the company's proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells. This reduces radiation exposure to normal tissues.
Cellectar is launching a phase 1 study to evaluate CLR 131 as treatment for children with Ewing sarcoma, rhabdomyosarcoma, osteosarcoma, neuroblastoma, high-grade glioma and lymphomas.
“[Ewing] sarcoma is the second most common bone malignancy among children and adolescents, and there are limited treatment options for patients who relapse or become refractive to therapy,” John Friend, MD, chief medical officer of Cellectar, said in a company-issued press release. “The orphan drug designation for [Ewing] sarcoma represents another important milestone for our CLR 131 pediatric program as we work to bring new options to patients suffering from rare cancers.”
A phase 2 study is assessing CLR 131 for treatment of relapsed or refractory multiple myeloma and multiple B-cell malignancies. A planned phase 1 study would evaluate the combination of CLR 131 and external beam radiation for patients with head and neck cancer.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.