FDA approved Novartis’ Afinitor® (everolimus) for the treatment of subependymal glial cell astrocytoma (SEGA) in patients who are not eligible for curative surgical resection. SEGA is a benign brain tumor associated with tuberous sclerosis, and primarily affects children and adolescents.
FDA clearance was based on studies confirming that treatment with Afinitor led to 32% of patients experiencing a reduction of 50% or more in the size of their largest SEGA tumor. In addition, none of the treated patients developed any new SEGAs while receiving Afinitor therapy. Everolimus is currently under review for the SEGA indication in other major markets, including the EU and Switzerland. Novartis says if approved in Europe the drug will be trade named as Votubia®
The firm points out that prior to FDA approval of Afinitor for the SEGA indication the only treatment option for the condition was brain surgery. Tuberous sclerosis is a genetic condition affecting about 25,000-40,000 people in the U.S., and causes the growth of SEGAs in up to 20% of patients, Novartis notes.
Afinitor is a once-daily oral inhibitor of mTOR (mammalian target of rapamycin). In the U.S. the drug is separately approved for the treatment of advanced renal cell carcinoma (RCC) after failure of therapy with either sunitinib or sorafenib. Similar clearance in the EU covers the use of Afinitor for the treatment of RCC in patients whose disease has progressed on or after VEGF-targeted therapy. Afinitor is being studied in range of tumor types, and Phase III trials are under way in breast cancer, gastric cancer, hepatocellular carcinoma, pancreatic neuroendocrine tumours (NET), and lymphoma. Novartis recently confirmed plans to submit Afinitor for the NET indication by year end.
Everolimus is separately available under the trade names Zortress™ and Certican® for use in the prevention of organ rejection after certain transplantation procedures. Novartis reported overall Afinitor sales for oncology indications were $67 million in the third quarter of 2010. Sales of everolimus as Zortres/Certican for transplantation procedures were $35 million during Q3 2010, up 19% on the same quarter the previous year.