FDA Approves the First Drug for Tuberous Sclerosis Tumors

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Good news for patients with subependymal giant cell astrocytoma (SEGA).  The US Food and Drug Administration (FDA) has granted the approval for everolimus (Afinitor, Novartis) as the first medication for children and adults with SEGA who require therapeutic intervention but are not candidates for curative surgical resection. 

SEGAs are slow-growing, benign brain tumors that occur in up to 20% of patients with tuberous sclerosis, a genetic disorder that affects about 25,000 to 40,000 people in the United States.  Patients with this disease currently have limited treatment options beyond surgical intervention.  

The approval was based on a priority review of a clinical study (n = 28) that showed that one third (32%) of patients treated with everolimus achieved more than 50% shrinkage of their largest SEGA tumor at 6 months. Of the 4 patients whose tumors returned after surgery, 3 met the endpoint with a 50% or greater reduction in tumor volume. 

The duration of response ranged from 3 months to 2.5 years (median, 9 months). Although not all SEGAs resolved completely, there is no new tumor during treatment. 

Adverse events appeared in 30% of the patients treated with everolimus.  These adverse events included mouth sores, upper respiratory tract infections, sinusitis, middle ear infections, and fever.  

Source: New England Journal of Medicine, 2010;363:1801-1811.

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