The Tuberous Sclerosis Alliance (TS Alliance) shared that the U.S. Food and Drug Administration has approved the drug Afinitor, manufactured by Novartis Pharmaceuticals, to treat partial-onset seizures in patients with tuberous sclerosis complex (TSC). TSC is a rare genetic disease that causes tumors to form in vital organs; it’s also the leading genetic cause of both epilepsy and autism.
“In TSC, 85 percent of individuals have seizures at some point in their lifetime. But very importantly and urgently, more than 60% experience intractable epilepsy, and this new indication for Afinitor means they now have a much-needed new treatment option,” said TS Alliance President & CEO Kari Luther Rosbeck. “Afinitor is the first mTOR inhibitor approved in the United States for the treatment of epilepsy and further demonstrates TSC as a model system for developing new therapies.”
Afinitor (everolimus) 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 TS Alliance played an important role in educating the TSC community about the EXIST-3 Phase III clinical trial of Afinitor, helping raise awareness of the opportunity to participate in the study. The success of this clinical trial ultimately led to this approved indication,” shared Steven L. Roberds, PhD, Chief Scientific Officer of the TS Alliance. “The TS Alliance-sponsored Patient Focused Drug Development meeting in June 2017 provided the opportunity for adults and parents of children with TSC to speak directly to the FDA about the unmet medical needs of TSC and how the TSC community weighed the risk and benefit of mTOR inhibitors to reduce the burden of refractory epilepsy in TSC.”