FDA grants orphan drug designation to Nerlynx for breast cancer brain metastases

In In The News by Barbara Jacoby

Source: FDA News

From: healio.com

The FDA has granted neratinib orphan drug designation for the treatment of patients with breast cancer and brain metastases, according to a press release from the drug’s manufacturer.

Neratinib (Nerlynx, Puma Biotechnology) — an irreversible pan-HER tyrosine kinase inhibitor — is approved for the extended adjuvant treatment of adults with HER2-overexpressed breast cancer, following adjuvant trastuzumab-based therapy.

About 20% to 25% of breast cancers overexpress HER2, which is a marker for more aggressive disease. Up to a quarter of these patients treated with trastuzumab experience disease recurrence.

“Receiving orphan drug designation from the FDA signifies our continued progress and commitment to developing treatments for patients with HER2-positive breast cancer,” Alan H. Auerbach, chairman, CEO and president of Puma, said in a press release. “Despite expanded treatment options for HER2-positive breast cancer, brain metastases in these patients represent a significant clinical challenge, as well as sources of morbidity and mortality for most of these patients. The blood-tumor penetrability of Nerlynx represents a potential treatment option for these underserved patients.”

In the phase 2 TBCRC 022 trial — published in Journal of Clinical Oncology in May — neratinib plus capecitabine demonstrated efficacy for the treatment of HER2-positive breast cancer brain metastases. Among 49 patients in the trial, researchers observed a composite central nervous system objective response rate of 49% (95% CI, 32-66) among those who had not previously received lapatinib (Tykerb, Novartis) and 33% (95% CI, 10-65) among those who had.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.


Freedman RA, et al. J Clin Oncol. 2019;doi:10.1200/JCO.18.01511.