Therapeutic efficacy of Everolimus

Hormone Receptor-Positive, HER2-Negative Breast Cancer (BOLERO-2 Study):

Compared with placebo + exemestane, everolimus + exemestane significantly improved investigator-assessed progression-free survival (median PFS: 7.8 months vs. 3.2 months) and achieved a higher objective response rate (12.6% vs. 1.7%). No statistically significant difference in overall survival was observed.

Neuroendocrine Tumors (NETs):

Pancreatic Neuroendocrine Tumors (RADIANT-3 Study): Compared with placebo, everolimus significantly improved progression-free survival (investigator-assessed median PFS: 11.0 months vs. 4.6 months).

Gastrointestinal or Pulmonary Neuroendocrine Tumors (RADIANT-4 Study): Compared with placebo, everolimus significantly improved independent-assessed progression-free survival (median PFS: 11.0 months vs. 3.9 months).

Functional Carcinoid Tumors (RADIANT-2 Study): Efficacy in locally advanced or metastatic functional carcinoid tumors was not demonstrated.

Renal Cell Carcinoma (RCC) (RECORD-1 Study):

Compared with placebo, everolimus significantly improved independent-assessed progression-free survival (median PFS: 4.9 months vs. 1.9 months). Overall survival could not be evaluated due to crossover to everolimus in the placebo group.

TSC-Associated Renal Angiomyolipoma (EXIST-2 Study):

The angiomyolipoma response rate (reduction ≥ 50%) in the everolimus group was significantly higher than that in the placebo group (41.8% vs. 0%). With a median long-term follow-up of 3.9 years, 58.0% of patients achieved a response.

TSC-Associated Subependymal Giant Cell Astrocytoma (SEGA):

EXIST-1 Study: The SEGA response rate (reduction ≥ 50%) in the everolimus group was significantly higher than that in the placebo group (35% vs. 0%). With a median long-term follow-up of 3.9 years, 58% of patients achieved a response.

Study 2485: An open-label study showed that 32% of patients had a SEGA volume reduction of ≥ 50% at 6 months.

TSC-Associated Partial Seizures (EXIST-3 Study):

Compared with placebo, AFINITOR DISPERZ significantly reduced seizure frequency in both the low (3-7 ng/mL) and high (9-15 ng/mL) concentration groups (median reduction rate: 29.3%, 39.6% vs. 14.9%), with higher response rates (seizure reduction ≥ 50%) (28.2%, 40.0% vs. 15.1%).

FDA，2022.02