The US Food and Drug Administration (FDA) has approved Novartis Afinitor (everolimus) to treat patients with non-cancerous kidney tumors associated with tuberous sclerosis complex (TSC).
The approval was based on the Phase III EXamining everolimus In a Study of TSC trial, which found that 42% of patients on everolimus experienced an angiomyolipoma response versus 0% of patients in the placebo arm.
Everolimus demonstrated superiority to placebo for both supportive efficacy outcomes measured, time to angiomyolipoma progression and skin lesion response rate, in the double-blind, randomized, placebo-controlled study.
Novartis Oncology president Herve Hoppenot said, "This approval further strengthens our commitment to address unmet needs in TSC as we continue to research everolimus and mTOR inhibition across other manifestations of the disease."
Defects in the TSC1 and/or TSC2 genes cause TSC. Due to the defects, mTOR activity is increased, which may lead to uncontrolled tumor cell growth and proliferation, blood vessel growth and altered cellular metabolism.
According to preclinical studies, by inhibiting mTOR activity in this signaling pathway, everolimus reduces cell proliferation and blood vessel growth, the company said.
