Novartis’ Afinitor (everolimus) has been approved by the Food and Drug Administration (FDA) for the treatment of patients with progressive, well-differentiated non-functional, neurodocrine tumors (NET) of gastrointestinal (GI) or lung origin with unresectable, locally advanced or metastatic disease.

Afinitor, an mTOR kinase inhibitor, was previously approved to treat progressive neuroendocrine tumors of pancreatic origin in adults with unresectable, locally advanced or metastatic disease. It is also indicated for renal cell carcinoma after failure of treatment with sunitinib or sorafenib; renal angiomyolipoma and tuberous sclerosis complex (TSC), not requiring immediate surgery; in postmenopausal women with advanced hormone receptor-positive, HER2-negative breast cancer (advanced HR+ BC) in combination with exemestane after failure of treatment with letrozole or anastrozole; and subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC) in adults and children who require therapeutic intervention but are not candidates for curative surgical resection. 

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The new approval is based on a multicenter, randomized (2:1), placebo controlled trial of everolimus vs. placebo. The efficacy outcome measure was progression-free survival (PFS) based on independent radiological assessment per RECIST. Median PFS was 11 months for the everolimus group and 3.9 months for the placebo group (HR 0.48 (95% CI: 0.35, 0.67), p <0.001, stratified log rank test).

The trial included a total of 302 patients, 64% of which were treated for >6 months and 39% for >12 months. Twenty-nine percent discontinued everolimus due to adverse reactions, with 3 patients dying due to cardiac failure, respiratory failure, and septic shock, respectively.

The most common adverse reactions were stomatitis, infections, diarrhea, peripheral edema, fatigue and rash.

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