The Food and Drug Administration has approved of the use of a drug called Afinitor Disperz. The FDA has approved it specifically for the use of treating brain tumors in young children who have a rare genetic disease. The drug was cleared under the FDA’s accelerated approval program.
Earlier this year, the Food and Drug Administration (FDA) approved a drug called Kalydeco. That was the first drug that the FDA has approved that was designed to help treat the cause of cystic fibrosis. Kalydeco was specifically approved for people who have cystic fibrosis and who are age six or older.
Now, the FDA has approved another drug that will help young patients who have a rare genetic disorder that causes brain tumors. This week, the FDA approved of a drug called Afinitor Disperz. It is a new formulation of the drug, and is created by Novartis. The drug has been specifically cleared to treat children who are three years old (or older) and who have inoperable tumors caused by tuberous sclerosis.
According to the Mayo Clinic, tuberous sclerosis is “a rare genetic disease that causes noncancerous (benign) lesions to grow in many parts of the body, such as the skin, brain and kidneys.”
The disease is often detected when a person is an infant or while the person is in childhood. Sometimes, the symptoms of the disease are mild, and a person remains undiagnosed until he or she reaches adulthood. Other times, the symptoms cause serious disabilities. There is no cure for tuberous sclerosis. Doctors cannot predict the course of the disease or the potential severity of it.
Tuberous sclerosis can cause a person to develop tumors in the brain, eyes, liver, lungs and other organs. This can lead to symptoms such as disabilities, seizures, and other problems. Obviously, having a tumor growing in one’s brain can lead to problems.
The FDA has now approved Afinitor Disperz for very young patients who have brain tumors that cannot be treated by surgery. The brain tumors are called subependymal giant cell astrocytoma. The drug has also been approved to treat lung, pancreatic, and kidney cancers.
The FDA approved the drug under its accelerated approval program. This process allows for new drugs to be approved based on preliminary clinical trials. Novartis will be required to conduct additional trials to confirm that the drug has the benefits it showed in the preliminary clinical trials.
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