The drug is approved as the first adjunctive therapy in the European Union (EU) for patients aged two years and older.
The EC’s decision marks the third TSC-related indication for Votubia in the EU, where it is also approved to treat subependymal giant cell astrocytoma (SEGA) and renal angiomyolipomas.
The approval was based on efficacy and safety data from a phase III study, which demonstrated that when used as an adjunctive therapy, seizure response rate (≥50% reduction) was significantly greater with Votubia at both low exposure (28.2%) and high exposure (40%) versus placebo (15.1%).
Novartis said the most common all-grade adverse events of any cause reported during the core phase at frequencies of greater than 15% in both Votubia treatment arms included stomatitis, diarrhea, nasopharyngitis, upper respiratory tract infection, and pyrexia.
Novartis Oncology CEO Bruno Strigini 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.
"This is a welcome advance and an important milestone in our ongoing commitment to improving care for this patient community."
Votubia inhibits the mammalian target of rapamycin (mTOR), a protein that regulates several cellular functions.
In the US, Votubia is approved as Afinitor tablets to treat adult patients with renal angiomyolipoma and TSC, not requiring immediate surgery.
Image: View of the Forum, Novartis HQ. Photo: courtesy of Novartis AG.