November 25, 2010
1 min read

FDA approves everolimus to treat subependymal giant cell astrocytoma

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The FDA has granted approval to Novartis to market the anticancer drug everolimus as a treatment for subependymal giant cell astrocytoma associated with tuberous sclerosis.

The indication is for patients with tuberous sclerosis who are not eligible for curative surgery. The disorder causes benign tumors to grow in the brain, as well as the eyes, lungs, liver, heart, skin and kidneys. Symptoms include learning and developmental disabilities, skin abnormalities, seizures, and lung and kidney disease.

Tuberous sclerosis affects 25,000 to 40,000 Americans annually, and subependymal giant cell astrocytomas are present in 6% to 9% of patients. Tuberous sclerosis can be fatal for patients who develop complications.

Everolimus (Afinitor) was first approved in March 2009 as second-line therapy for patients with kidney cancer who are refractory for sunitinib (Sutent, CPPI CV) or sorafenib (Nexavar, Bayer). The drug was approved for this new indication under FDA’s accelerated approval program. Novartis is required to collect additional data showing long-term efficacy and safety, but the FDA said the program allows patients earlier access to promising new or existing drugs while confirmatory clinical trials are ongoing.

FDA granted approval based on results from a single study of 28 patients. At 6 months, 32% of patients had a more than 50% reduction in tumor volume of their largest subependymal giant cell astrocytoma tumor lesion. Median duration of response was 266 days.

Four patients saw their tumors return after surgery. Three of those patients saw tumor volume shrink by more than 50% after receiving everolimus.

The most common reported adverse events included upper respiratory tract infections, sinus and ear infections, mouth sores and fever.