FDA approves Novartis drug for rare disease
The Food and Drug Administration said Monday it approved Novartis' drug Afinitor to treat some of the tumors caused by a rare genetic disorder called tuberous sclerosis.
Tuberous sclerosis causes tumors in the brain, eyes, lungs, liver and other organs, leading to learning disabilities, seizures and other problems. The tumors are not cancerous, but the disorder -- which affects 25,000 to 40,000 people in the U.S., according to a Novartis spokeswoman -- can be fatal for patients who develop complications with tumor growth in the brain.
The agency approved Afinitor for the 6 percent to 9 percent of tuberous sclerosis patients who have benign brain tumors called subependymal giant cell astrocytoma that can't be treated with surgery, the FDA said.
Afinitor is already approved as a treatment for advanced renal cell carcinoma.
The FDA approved the new indication based on a 28-patient study in which nine patients experienced a 50 percent reduction in the size of their tumors.
Afinitor was cleared under the FDA's accelerated approval program, which allows approval of new drugs based on preliminary clinical trial results. Companies are required to conduct additional studies to confirm the benefits of their drugs.
Nov 03, 2010