FDA backs Afinitor Disperz to treat TSC-associated partial-onset seizures

The US Food and Drug Administration has approved Afinitor Disperz (everolimus tablets for oral suspension), for the adjunctive treatment of adult and pediatric patients aged two years and older with tuberous sclerosis complex (TSC)-associated partial-onset seizures.

Swiss pharma giant Novartis’ (NOVN: VX) Afinitor Disperz is now the first approved pharmacologic therapy in the USA specifically indicated for the treatment of this condition.

TSC is a rare genetic disorder affecting up to one million people worldwide. Around 85% of individuals with TSC are affected by epilepsy, and uncontrolled seizures associated with TSC can be debilitating for patients. More than 60% of TSC patients who experience seizures stop responding to available anti-epileptic therapies. EXIST-3 is the first Phase III study to demonstrate the significant benefit of adjunctive Afinitor Disperz in the treatment of patients with TSC-associated partial-onset seizures. Furthermore, Afinitor is the only approved non-surgical option indicated for treating TSC-associated non-cancerous brain tumors (subependymal giant cell astrocytoma, or SEGA) and TSC-associated kidney tumors (renal angiomyolipoma).