Novartis’ Afinitor drug reduces seizures in tuberous sclerosis complex patients

TSC is a rare genetic disorder affecting about one million people globally, and Afinitor is the only approved non-surgical option indicated to treat non-cancerous brain and kidney tumours in several patients with the condition.

Afinitor inhibits the mammalian target of rapamycin (mTOR), a protein which regulates several cellular functions.

The EXIST-3 study found that the percentage reduction from baseline in seizure frequency was significantly greater among patients randomized to receive Afinitor in the low exposure arm (LE; 29.3%) and high exposure arm (HE; 39.6%) compared to placebo (14.9%).

Novartis said seizure response rate (>=50% reduction) was also significantly greater with Afinitor LE (28.2% and HE (40%) vs placebo (15.1%).

The most common adverse events reported in those taking Afinitor LE/HE vs placebo included stomatitis, mouth ulceration, and diarrhoea.

Novartis oncology development and medical affairs global head Alessandro Riva said: "There has been a long-standing need to find a treatment option for TSC patients that provides control of treatment-resistant seizures and we are encouraged that data from the EXIST-3 study show everolimus may have this potential."

Earlier this year, the US Food and Drug Administration approved the use of Afinitor to treat adult patients with neuroendocrine tumors of gastrointestinal or lung origin.

In the European Union, Afinitor is approved as Votubia to treat adult patients with renal angiomyolipoma associated with TSC who are at risk of complications without immediate surgery.

Image: Afinitor Product Shot. Photo: courtesy of Novartis.