The new formulation, Afinitor Disperz, was cleared to treat children as young as 1 year old who have inoperable tumors caused by 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 they can be fatal for patients who develop complications from tumor growth in the brain.
The drug was previously approved for children 3 years old and up.
The FDA approved Afinitor Disperz for very young patients who have brain tumors called subependymal giant cell astrocytoma that can't be treated with surgery.
Afinitor is also approved to treat various types of lung, pancreatic and kidney cancers. The drug blocks a protein that plays a role in uncontrolled tumor growth.
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.