FDA approves drug for treating tumors associated with rare genetic disorder

SILVER SPRING, Md. The Food and Drug Administration has approved a cancer drug for treating tumors associated with a rare genetic disorder.

The agency announced Monday the approval of Novartis’ Afinitor (everolimus) for patients who develop slow-growing tumors known as subependymal giant-cell astrocytoma, or SEGA, related to the disease tuberous sclerosis. TS causes benign tumors to grow in the brain and other parts of the body, and results in a variety of symptoms, including learning disabilities, skin abnormalities, seizures and kidney disease.

Afinitor was approved last year for kidney cancer patients for whom such treatments as Pfizer’s Sutent (sunitinib) and Bayer HealthCare and Onyx Pharmaceuticals' Nexavar didn’t work. It also is approved under another name, Zortress, for preventing rejection of transplanted organs.

“Patients with this disease currently have limited treatment options beyond surgical intervention,” FDA Office of Oncology Drug Products director Richard Pazdur stated. “It is important for research to continue in rare diseases where patients have few or no existing drug treatment options.”