The European Commission has expanded the approved uses of Novartis' Votubia to include adjunctive treatment of patients aged two years and older whose refractory partial-onset seizures, with or without secondary generalisation, are associated with tuberous sclerosis complex.
Affecting up to one million people worldwide, TSC is a rare genetic disorder that can lead to non-cancerous tumours being formed in vital organs, as well as linked disorders such as epilepsy, autism, cognitive impairment, behavioural problems and psychiatric disorders.
The approval makes Votubia (everolimus) the first adjunctive treatment approved in the region specifically for partial-onset seizures in children and adults with TSC, which could help address an unmet need given that up to 60 percent of patients become unresponsive to available anti-epileptic therapies.
The nod was based on efficacy and safety data from a pivotal Phase III study (EXIST-3), which found that when used as an adjunctive therapy, seizure response rate (≥50 percent reduction) was significantly greater with Novartis drug at both low exposure (28.2 percent) and high exposure (40.0 percent) versus placebo (15.1 percent).
On the safety side, the most common all-grade adverse events of any cause reported during the core phase at frequencies of greater than 15 percent in both Votubia treatment arms included stomatitis, diarrhoea, fever, nasopharyngitis, and upper respiratory tract infection, the firm said.
"With this latest approval of Votubia in the EU, patients with TSC suffering from refractory partial-onset seizures – one of the most debilitating manifestations of TSC – now have a new therapeutic option to address a critical unmet need," said Bruno Strigini, chief executive of Novartis Oncology. "This is a welcome advance and an important milestone in our ongoing commitment to improving care for this patient community."
In the EU, Votubia is also cleared for the treatment of adult patients with renal angiomyolipoma associated with TSC who are at risk of complications but who do not require immediate surgery, and patients with subependymal giant cell astrocytoma (SEGA) associated with TSC who require therapeutic intervention but are not amenable to surgery.