FDA Approves Novartis’ Afinitor DISPERZ to Treat TSC-Associated Partial-Onset Seizures

Rare Daily Staff    

The U.S. Food and Drug Administration approved Novartis’ Afinitor DISPERZ for the adjunctive treatment of adult and pediatric patients aged two years and older with tuberous sclerosis complex-associated partial-onset seizures.

Afinitor DSPERZ is the first approved pharmacologic therapy in the U.S. specifically indicated for the treatment of this condition. The medication is also the only approved non-surgical option indicated for treating TSC-associated non-cancerous brain tumors and TSC-associated kidney tumors.

Tuberous sclerosis complex is a rare genetic disorder that affect up to 1 million people worldwide. It is a multi-organ disease with widely variable expression. It may cause non-cancerous tumors to form in vital organs including the brain, kidney, heart, lungs, and skin, as well as resulting disorders such as epilepsy, autism, cognitive impairment, behavioral problems, and psychiatric disorders.  About 85 percent of people with TSC are affected by epilepsy. More than 60 percent of TSC patients who experience seizures stop responding to available anti-epileptic therapies.

“We are pleased that this latest approval for Afinitor DISPERZ in the U.S. will make an important difference to patients with tuberous sclerosis complex who experience partial-onset seizures, one of the most debilitating manifestations of TSC,” said Ameet Mallik, Executive Vice President, Novartis Oncology U.S. “This is a welcome advance that reinforces the commitment of Novartis to patients with rare diseases.”

The FDA approval of Afinitor DISPERZ was based on efficacy and safety data from a pivotal Phase III study, which found that when used as an adjunctive therapy, Afinitor DISPERZ significantly reduced the frequency of treatment-resistant seizures associated with TSC compared to placebo.

April 11, 2018
Photo: Ameet Mallik, Executive Vice President, Novartis Oncology U.S.