FDA Approves Everolimus for GI, Lung Neuroendocrine Tumors

A new treatment was approved today by the FDA for the treatment of progressive, non-functional neuroendocrine tumors (NET) of gastrointestinal (GI) or lung origin.

The approval of everolimus (Afinitor) followed a randomized (2:1) clinical trial with 302 patients that suffered from NET of GI or lung origin and showed evidence of disease progression within 6 months prior to randomization.

Patients in the trial were administered either 10 mg of Afinitor orally and daily with best supportive care (BSC) or a placebo and BSC.

Progression-free survival (PFS) was the major efficacy outcome measure based on independent radiological assessment per RECIST.

The results of the study showed patients administered Afinitor plus BSC had a median PFS of 11 months, while the placebo group plus BSC was 3.9 months [HR 0.48 (95% CI: 0.35, 0.67), p <0.001, stratified log rank test].

The overall response rate in the Afinitor group was 2% compared to 1% in the placebo group.

When researchers reached the planned interim analysis, they found that there was not a statistically significant difference in overall survival rate between the 2 groups.

When the safety data was evaluated for 300 participants who received at least 1 dose of Afinitor, it showed a median exposure duration of 9.3 months. About 64% of patients were treated for greater than or equal to 6 months, and 39% were treated for 12 months.

Adverse reactions in 29% of patients led to a discontinuation, dose reduction, or delay of Afinitor. Three fatal adverse events occurred: cardiac failure, respiratory failure, and septic shock, which effected 42% of participants.

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

The common laboratory abnormalities were elevated aspartate transaminase (AST), fasting hyperglycemia, anemia, hypercholesterolemia, and lymphopenia.