FDA Clears Everolimus Tablets for Seizures in Tuberous Sclerosis

Megan Brooks


April 11, 2018

The US Food and Drug Administration (FDA) has approved everolimus tablets for oral suspension (Afinitor Disperz, Novartis) for the adjunctive treatment of adults and children aged 2 years and older with tuberous sclerosis complex (TSC)–associated partial-onset seizures.

Everolimus tablets for oral suspension become the first approved pharmacologic therapy in the United States specifically indicated for the treatment of this condition, the company said in a news release.

In the EXIST-3 (EXamining everolimus In a Study of TSC) phase 3 study, everolimus tablets for oral suspension, when used as an adjunctive therapy, significantly reduced the frequency of treatment-resistant seizures associated with TSC compared with placebo.

Response rates, defined as a 50% or greater reduction in seizures, were also greater with everolimus than with placebo. The most common adverse events seen with everolimus included stomatitis, diarrhea, nasopharyngitis, upper respiratory tract infection, and pyrexia.

TSC is a rare genetic disorder affecting up to 1 million people worldwide. More than 60% of patients with TSC experience seizures that have become resistant to available antiepileptic therapies.

TSC is caused by defects in the TSC1 and TSC2 genes that negatively control the mammalian target of rapamycin (mTOR). Everolimus inhibits mTOR. In animal models of mTOR dysregulation, treatment with an mTOR inhibitor resulted in prolonged survival, seizure suppression, prevention of new-onset seizures, and prevention of premature death.

Everolimus was granted accelerated FDA approval in 2010 to treat subependymal giant cell astrocytoma in patients with TSC. The agency approved a pediatric dosage form of everolimus in 2012. 

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