FDA approves Afinitor Disperz for tuberous sclerosis complex.- Novartis.

The FDA has approved Afinitor Disperz (everolimus tablets for oral suspension), manufactured by Novartis Pharmaceuticals, to treat partial-onset seizures in patients with tuberous sclerosis complex (TSC). In TSC, 85 percent of individuals have seizures at some point in their lifetime. But more than 60% experience intractable epilepsy, and this new indication for Afinitor means they now have a new treatment option. Afinitor was previously approved by the FDA for the treatment of adult and pediatric patients aged 1 year and older with TSC who have subependymal giant cell astrocytoma (SEGA) and adults with renal angiomyolipomas.

The FDA approval of Afinitor Disperz was based on efficacy and safety data from a pivotal Phase III study, EXIST-3 (EXamining everolimus In a Study of TSC), which found that when used as an adjunctive therapy, Afinitor Disperz significantly reduced the frequency of treatment-resistant seizures associated with TSC compared to placebo. The median percentage reduction from baseline in seizure frequency was significantly greater among patients randomized to Afinitor Disperz low exposure (LE; 29.3%) and high exposure (HE; 39.6%) vs placebo (14.9%). Seizure response rate (at least a 50% reduction) was also greater with Afinitor LE (28.2%) and HE (40.0%) vs placebo (15.1%). The most common all-grade adverse events of any cause reported during the core phase at frequencies of more than 15% in the Afinitor Disperz LE/HE arms included stomatitis, diarrhea, nasopharyngitis, upper respiratory tract infection, and pyrexia