The FDA accelerated the approval of the oral mTOR kinase inhibitor everolimus (Afinitor; Novartis) for the treatment of kidney tumors (ie, renal angiomyolipomas) not requiring immediate surgery in patients with tuberous sclerosis complex (TSC). This is the first drug approved specifically by the FDA for this patient population.
TSC is a rare genetic disease causing noncancerous tumors in the kidney, brain, and other organs. There are approximately 40,000 patients with TSC in the United States, of whom 70% or 80% develop kidney problems.
The FDA approval was based on the double-blind, randomized, placebo-controlled, international, multicenter phase 3 EXIST-2 (Examining Everolimus in a Study of TSC) trial, which showed a 42% angiomyolipoma response rate with everolimus compared with 0% response with placebo.
“Today marked an important step for the TSC community, as Afinitor is now the only approved medicine to reduce kidney tumor burden in these patients,” said John Bissler, MD, Clark D. West Chair of Nephropathy, Cincinnati Children’s Hospital.
The most common side effects with everolimus included inflamed/sour mouth, nausea/vomiting, skin problems, cough, headache, diarrhea, abdominal pain, joint pain/swelling, and upper respiratory infection.
Everolimus is already approved as an orphan drug for advanced renal-cell carcinoma after failure of treatment with sunitinib (Sutent; Pfizer) or with sorafenib (Nexavar; Bayer/ Onyx), for progressive neuroendocrine tumors of pancreatic origin in unresectable, locally advanced or metastatic disease, and for subependymal giant-cell astrocytoma associated with tuberous sclerosis. (April 26, 2012)
