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Pediatric Osteosarcoma Drug Receives Orphan Drug Designation

The Food and Drug Administration (FDA), the agency tasked with overseeing public health in the U.S., has given this classification to CLR 131, a drug designed by Cellectar Biosciences for the treatment of a rare type of cancer affecting children, pediatric osteosarcoma. The Orphan Drug Designation is a special status granted to “a drug or [...]

The Food and Drug Administration (FDA), the agency tasked with overseeing public health in the U.S., has given this classification to CLR 131, a drug designed by Cellectar Biosciences for the treatment of a rare type of cancer affecting children, pediatric osteosarcoma.

The Orphan Drug Designation is a special status granted to “a drug or biological product to treat a rare disease or condition upon request of a sponsor”. This comes with a number of incentives, namely a market exclusivity period of seven years, increased support and assistance from the FDA, the possibility of receiving tax credits for research, grants and a waiver of the New Drug Application user fee.

John Friend, M.D., chief medical officer of Cellectar said in a press release that “this orphan designation for osteosarcoma is the fourth such designation granted by the FDA to CLR 131 for the treatment of rare pediatric cancers in the last six months, and we look forward to evaluating CLR 131 in these deadly and underserved diseases”.

Osteosarcoma is a type of cancer that affects the formation of mesenchymal, a connecting tissue found throughout the body, especially in bones, muscle, and cartilages. When the disease takes over, it damages the bone tissue, making it more brittle than normal bones.

CLR131 is an investigational radioiodinated PDC therapy that helps with better targeting the radiation on the malignant tumor cells and minimizes exposure to normal tissue.

While incidence rate for osteosarcoma is quite low, about 4.4 cases per 1 million per year, current treatment options are not always effective. If diagnosed in initial stages, the cure rate can go as up as 70%, unfortunately, this is not always the case. Five-year overall survival rates for patients who develop metastasis is approximately 20%. Those who experience disease progression or recurrence have a survival chance that is less than 30%.

Trials so far are still in the preliminary phases, but the results are quite promising especially since all doses and regimens have been deemed safe and well tolerated by patients

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