FDA Grants Orphan Drug Designation to Nerlynx® for Breast Cancer-Related Brain Metastases

The U.S. Food and Drug Administration (FDA) granted orphan drug designation to Nerlynx® (neratinib) for patients with breast cancer with brain metastases.

Neratinib is a tyrosine kinase inhibitor that is FDA-approved for the extended adjuvant treatment of adults with human epidermal growth factor receptor 2 (HER2)-positive breast cancer following adjuvant trastuzumab-based therapy.

Response observed in phase II study

In the phase II TBCRC 022 trial, patients received neratinib 240 mg orally once per day plus capecitabine 750 mg/m² twice per day for 14 days, then seven days off.

This combination demonstrated efficacy for the treatment of HER2+ breast cancer-related brain metastases. Among 49 patients, there was a composite central nervous system objective response rate of 49% (95% CI, 32-66) in those who had not previously received lapatinib and 33% (95% CI, 10-65) in those who had. Median progression-free survival was 5.5 and 3.1 months, respectively, while median survival was 13.3 and 15.1 months, respectively.

Diarrhea was the most common grade 3 adverse event, occurring in 29% of patients who did and did not receive previous lapatinib.