Everolimus treats patients aged 3 years and older who require therapeutic intervention but are not amenable to surgery.
Everolimus targets mTOR, a protein that acts as a regulator of tumor cell division, blood vessel growth and cell metabolism.
The EC approval follows Committee for Medicinal Products for Human Use (CHMP) positive opinion based on a open-label, prospective, single-arm, Phase II study of 28 patients showing SEGA tumor reduction in patients with TSC.
An additional placebo-controlled phase III study examining the patient population met its primary endpoint of SEGA response rate.