According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals, Inc. has announced recently that its experimental diagnostic 64Cu-SARTATE™ has earned Rare Pediatric Disease designation from the US Food and Drug Administration (FDA). This diagnostic is being developed for the treatment of neuroblastoma (as an imaging agent), a rare cancer that primarily impacts children. Clarity is focused on the development of therapies for serious, life-threatening illnesses.
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 reserved for experimental therapies and products that are in development for the treatment of rare diseases that primarily affect people age 18 or younger. A rare disease is defined as any illness that affects less than 200,000 people in the US. If the product in question is ultimately approved for public use by the FDA, the company is then given a Priority Review voucher. This voucher allows for a shortened review period lasting just six months for a future product. The company can also sell or trade the voucher to another company.
About 64Cu-SARTATE and 67Cu-SARTATE
The company was also recently awarded the same designation for 67Cu-SARTATE, a therapy being developed for neuroblastoma. 64Cu-SARTATE is designed as an imaging agent that can detect the presence of the SSTR2 receptor on the cancer cells. 67Cu-SARTATE is being developed alongside it as a treatment; they both contain forms of copper that produce radiation. Learn more about these experimental medical products here.