Novartis’ Afinitor Disperz has become the first medicine to be specifically approved by US regulators to treat patients aged two years and above with tuberous sclerosis complex (TSC)-associated partial-onset seizures.
TSC is a rare genetic disorder affecting up to one million people around the globe. Around 85 percent of individuals with TSC are affected by epilepsy, and uncontrolled seizures associated with TSC can be debilitating for patients.
The Swiss drug giant said the new approval for Afinitor Disperz (everolimus) addresses unmet need as more than 60 percent of patients with TSC suffer from seizures that have become resistant to available anti-epileptic therapies.
In clinical trials, the oral mTOR pathway inhibitor significantly reduced the frequency of treatment-resistant seizures associated with TSC compared to placebo when used as adjunctive therapy.
The median percentage reduction from baseline in seizure frequency was significantly greater among patients randomized to receive Afinitor Disperz, with 29.3 percent for the low exposure group and 39.6 percent for the high exposure group versus 14.9 percent for placebo.
"We are pleased that this latest approval for Afinitor Disperz in the US will make an important difference to patients with tuberous sclerosis complex who experience partial-onset seizures, one of the most debilitating manifestations of TSC," said Ameet Mallik, Executive Vice President, Novartis Oncology US.
The approval marks the third TSC-related indication cleared for Afinitor in the US, with the drug already on the market to treat certain patients with subependymal giant cell astrocytoma (SEGA) and renal angiomyolipoma.