FDA Grants QSAM Rare Pediatric Disease Designation for Osteosarcoma Therapy

The U.S. Food and Drug Administration granted QSAM Biosciences Rare Pediatric Disease Designation for its experimental therapy CycloSam (Samarium-153-DOTMP) for the treatment of osteosarcoma, a form of bone cancer that afflicts mostly children and young adults.

Osteosarcoma is the most common form of bone cancer in children and young adults with primary high-grade bone malignancy. There have been few advancements over the last 40 years for this debilitating and often deadly disease, with treatment often resulting in limb amputation. Accordingly, there is a large unmet market need for a better treatment that is more efficacious against pediatric osteosarcoma and better tolerated by patients.

CycloSam is a nuclear technology that uses low specific activity Samarium-153 (resulting in far less europium impurity) and DOTMP, a chelator which is believed to eliminate off-target migration and targets sites of high bone turn over, making it, in management’s opinion, an ideal agent to treat primary and secondary bone cancers. Since CycloSam delivers targeted radiation selectively to the skeletal system, it is also believed to be an effective agent to perform bone marrow ablation as pre-conditioning for bone marrow transplantation, and in procedures to reduce external beam radiation to bone tumors. The experimental therapy uses an FDA approved radioisotope combined with a novel chelant that has demonstrated preliminary increased efficacy and decreased side effects in animal models and veterinary treatment of bone cancer in dogs.

“Combined with the orphan designation for osteosarcoma that we received last year from the FDA, the Rare Pediatric Disease Designation may allow QSAM to potentially bring CycloSam to market more rapidly through additional incentives and eligibilities that ultimately help these young patients for whom there is currently little hope,” said Douglas Baum, CEO of QSAM.

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children aged 18 years or younger and impact fewer than 200,000 people in the United States. If a New Drug Application in the United States for CycloSam is approved, QSAM may be eligible to receive a Priority Review Voucher from the FDA.

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, Mirum Pharmaceuticals sold its voucher for $110 million.

Author: Rare Daily Staff