FDA Grants Cellectar Biosciences Rare Pediatric Disease Designation for Experimental Osteosarcoma Drug

September 19, 2018

Rare Daily Staff

The U. S. Food and Drug Administration has granted Rare Pediatric Disease designation to Cellectar Biosciences for its experimental drug CLR 131 to treat rare pediatric cancer, osteosarcoma.

Osteosarcoma is a type of cancer that starts in the bones. It is believed to derive from bone-forming mesenchymal or connective tissue and cells. It is the most commonly diagnosed primary bone malignancy among children and adolescents.

CLR 131 is a small-molecule, targeted phospholipid drug conjugate designed to deliver cytotoxic radiation directly and selectively to cancer cells. CLR 131 is Cellectar’s lead therapeutic PDC product candidate and is currently being evaluated in multiple clinical studies.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes CLR 131 eligible for a Rare Pediatric Disease Priority Review Voucher upon approval of the therapy by the FDA.

This year the company secured Rare Pediatric Drug designations for CLR 131 in four pediatric cancers, including neuroblastoma, rhabdomyosarcoma, Ewing’s Sarcoma and osteosarcoma. If any of these indications achieve approval, the company could secure a Rare Pediatric Disease Priority Review Voucher.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Ultragenyx and Kyowa Hakko Kirin sold their Priority Review Voucher for $80.6 million.

“CLR 131 has demonstrated promise as an anticancer agent in preclinical and clinical settings, and we are working now to establish its impact on various rare and deadly pediatric cancers,” said John Friend, Cellectar Biosciences chief medical officer. “Cellectar is pleased to have the opportunity to work closely with the FDA on our planned Phase 1 trial for these indications and we remain committed to advancing the pediatric programs as rapidly as possible.”

September 19, 2018
Photo: John Friend, Cellectar Biosciences chief medical officer


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