The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Afinitor® (everolimus) for the treatment of a benign brain tumor—subependymal giant cell astrocytoma associated with tuberous sclerosis—in children and adults who require treatment but are not candidates for surgical removal of the tumor.
Tuberous sclerosis is a rare genetic condition that causes benign tumors to grow in the brain and other organs. Subependymal giant cell astrocytoma (SEGA) is a type of benign brain tumor that may develop in people with tuberous sclerosis.
Afinitor is an oral targeted therapy that works by inhibiting a protein known as the mammalian target of rapamycin (mTOR). The mTOR protein plays an important role in regulating cancer cell division and blood vessel growth. Afinitor was originally approved in 2009 for the treatment of selected patients with advanced renal cell (kidney) cancer.
The safety and efficacy of Afinitor for SEGA associated with tuberous sclerosis was evaluated in a study of 28 patients. All of the patients had evidence of SEGA growth prior to the start of the study, and all were treated with Afinitor once daily. Patients ranged in age from three to 34 years, with a median age of 11 years.
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- At six months, nine of the 28 patients (32%) had a 50% or greater reduction in the volume of their largest SEGA lesion. Responses lasted for a median of 266 days.
- Three of the four patients with a history of prior surgery had a greater than 50% reduction in tumor volume.
- All patients experienced at least one adverse side effect. The most common adverse effects were mouth sores, upper respiratory infections, sinusitis, ear infections, and fever.
Based on these results, the FDA granted accelerated approval to Afinitor for the treatment of patients with SEGA associated with tuberous sclerosis who require therapy but are not candidates for surgical resection. The FDA’s accelerated approval program provides patients with access to treatments that may not yet have a confirmed clinical benefit but that address previously unmet medical needs.
A Phase III trial is currently underway to further evaluate Afinitor for SEGA associated with tuberous sclerosis.
Reference: U.S. Food and Drug Administration. Everolimus (Accelerated Approval). Accessed November 2, 2010.