On Monday, the Food and Drug Administration gave approval to Novartis' drug Afinitor to be used in the treatment of certain tumours that are thought to be caused by a rare genetic disorder called tuberous sclerosis.
The FDA said Afinitor drug could be used to treat 6-9% of tuberous sclerosis patients suffering from benign brain tumours called subependymal giant cell astrocytoma (SEGA) that is untreatable by an operation.
Tuberous sclerosis leads to the formation of tumours in the brain, eyes, lungs, liver, and other parts of the body. A person having this illness usually suffers from learning disabilities, seizures, and other complications.
Although the tumours do not become cancerous, the disorder can be deadly for those who suffer from complications due to tumour growth in the brain.
People having late-stage renal cell carcinoma are already being treated with Afinitor drug.
Mostly children and youngsters are at increased risk of tuberous sclerosis and presently limited treatment options are available for patients having this disorder.
The drug was approved after it was found in a study that it reduced the size of the largest SEGA tumour in one-third of 28 patients who were included in the trial.
According to Novartis, around 25,000 to 40,000 people in America are diagnosed with tuberous sclerosis, and 20% of them have the SEGA tumours.