WASHINGTON, April 26 U.S. health regulators
approved on Thursday Novartis AG's Afinitor for the
treatment of non-cancerous kidney tumors in patients with a rare
genetic disease known as tuberous sclerosis complex.
The approval from the Food and Drug Administration makes
Afinitor the first drug available specifically for non-cancerous
tumors that do not require immediate surgery.
Shares of Swiss-based Novartis closed 0.2 percent higher in
Europe earlier on Thursday.
Tuberous sclerosis complex, or TSC, causes multiple tumors
in both kidneys that compress normal tissue, leading to kidney
failure and bleeding.
The disorder affects about 40,000 people in the United
States, 70-80 percent of whom develop kidney problems.
Novartis' treatment, known generically as everolimus, is a
pill taken once daily that blocks the activity of a protein that
plays a critical role in the development and growth of
"This approval underscores the FDA's commitment to the
development of drugs for rare diseases with significant unmet
medical needs," said Dr. Richard Pazdur, director of hematology
and oncology products within FDA's Center for Drug Evaluation
"It also represents another example of where an
understanding of a disease's underlying biology can lead to more
selective drug development."
Novartis released data from a late-stage study last
September showing that Afinitor helps some people with the
non-cancerous kidney tumors.
The 118-patient study found that 42 percent of TSC sufferers
can experience responses including reduced tumor size and an
absence of new tumors after taking the drug.
The Swiss drugmaker is increasingly focusing on specialist
drugs to help protect its profitability, at a time when
top-selling drugs such as the blood-pressure treatment Diovan
face generic competition.