Experimental Treatment for Neuroblastoma Earns Rare Pediatric Disease Designation

According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals has recently announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to its product candidate Cu-SARTATE™. This therapy is being tested as a treatment for neuroblastoma, rare form of cancer that disproportionately impacts children. The product has previously earned Orphan Drug designation from the FDA as well.

About Neuroblastoma

Neuroblastoma is a type of cancer that appears in nerve tissue. Children are more frequently affected than adults. The direct cause of neuroblastoma is not known in most cases; in a small number of patients, an inherited mutation ALK gene has been implicated, but this only explains around two percent of cases at the most. Symptoms of neuroblastoma include a lump in the affected area which may be blue in color, bone and joint pain, loss of appetite, fatigue, and fever. Other symptoms vary depending on where the tumor is located; it typically originates in the adrenal glands but can also appear on the spine, neck, abdomen, or chest. Treatment approaches include immunotherapy, radiation therapy, chemotherapy, surgery, and stem cell transplant. Outcomes vary considerably; the five year survival rate is 68 percent for patients between age one and fourteen. To learn more about neuroblastoma, click here.

About Rare Pediatric Disease Designation

Rare Pediatric Disease designation is allotted to experimental medications in development that are intended to treat diseases or disorders that primarily impact people aged 18 years or younger that affect less than 200,000 people in the US. If a product with this designation ultimately gains approval for use by the public from the FDA, the developing company may be eligible for a Priority Review voucher. This voucher can be used for a future product in the company pipeline in order to receive a New Drug Application review in a more rapid six month period. Alternatively, these vouchers can be sold.

Though there are some effective therapies for neuroblastoma, options remain limited for patients with advanced, high risk disease. The awarding of these designation to Cu-SARTATE highlights the urgent need for more effective treatments for high risk patients. The drug is currently being evaluated in a phase 1/2 clinical trial.


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