FDA granted accelerated approval to Novartis’ Afinitor® (everolimus) tablets for the treatment of adult patients with renal angiomyolipomas and tuberous sclerosis (TSC) complex who don’t need immediate surgery. The firm says the mTor inhibitor treatment is the first to be cleared for use in this patient population.
TSC is a genetic disorder that can cause noncancerous tumors to form in vital organs, and up to 80% of patients will develop angiomylopiomas that can lead to severe kidney damage and require surgery. Skin lesions in addition occur in over 90% of TSC patients.
U.S. approval of everolimus for the TSC and angiomyolipoma indication was based on data from the placebo-controlled Phase III Exist-2 study, which demonstrated a 42% angiomyolipoma response rate among patients treated with Afinitor compared with 0% for placebo-treated patients. Afinitor therapy also significantly prolonged time to angiomyelipoma progression. There was in addition a 26% skin lesion response rate (a 50% or more improvement) among the 97% of trial patients who presented with skin lesions.
Afinitor has previously been approved in the U.S. and other markets for the treatment of adult patients with advanced renal cell carcinoma, progressive neuroendocrine tumors of pancreatic origin, and for treating adult adn pediatric patients with subependymal giant cell astrocytoma (SEGA) associated with TSC who aren’t suitable for surgical intervention. Everolimus is in addition marketed in the U.S. and other countries under the trade names Zortress and Certican for use in the prevention of organ rejection in adult kidney and heart transplant patients.
The drug is undergoing Phase III development in combination with exemestane as a treatment for postmenopausal women with ER+Her2- advanced breast cancer.