The U.S. Food and Drug Administration (FDA) have just approved a drug called Afinitor DISPERZ for treating tuberous sclerosis complex-associated partial onset seizures, reports PR Newswire. This makes it the first drug therapy approved in the US to treat this disease.
Tuberous sclerosis complex (TSC) is a rare genetic disease that affects approximately 1 in every 6,000 people. It causes tumours to form in various body parts. Although the tumours are usually benign, or non-cancerous, they can still cause serious problems in the areas affected. Since the tumours can form anywhere, the effects of the disease are diverse, and can include seizures, breathing difficulties, learning difficulties, kidney problems, fluid build-up in the brain, and skin problems amongst others. Some people only have a small number of mild symptoms, while others have many serious ones.
TSC is caused by mutations in the TSC1 or TSC2 genes. These genes regulate cell growth, but people with TSC have gene mutations that prevent them from fulfilling the function resulting in tumour growth. There is not currently a cure for TSC, mTOR inhibitors, which prevent tumour growth, are expected to be a source of future treatments. The newly approved drug, Afinitor DISPERZ, uses the active ingredient everolimus, which is a type of mTOR inhibitor.
Afinitor, known in some countries as Votubia, decreases the frequency of seizures in patients with TSC. It will address an important unmet patient need, since 85% of people with TSC also have epilepsy, and of these, over 60% of patients with seizures stop responding to available medicines. The drug has previously been approved in over thirty other countries for seizures in TSC patients, and now the Food and Drug Administration has also approved it for use in the US on patients aged two years and older.
Afinitor is also available in over 95 countries, including some EU countries and the US, for treating patients with brain tumours and kidney tumours caused by TSC.
The FDA’s decision to approve the drug will make it available to many patients with TSC in the US who urgently need more options for controlling debilitating seizures.