Puma Biotechnology's Kinase Inhibitor NERLYNX Granted FDA Orphan Drug Designation for HER2-Positive Breast Cancer with Brain Metastases

On September 2, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that biotechnology company Puma Biotechnology (“Puma”) announced that its kinase inhibitor NERLYNX had received FDA Orphan Drug Designation for the treatment of patients with HER2-positive breast cancer brain metastases.

In the United States, drugs used to treat rare diseases affecting fewer than 200,000 people are granted orphan drug designation by the FDA. Orphan drugs are eligible for several incentives, including waivers of clinical trial fees, tax credits, and waivers of user fees.

Breast cancer is the most prevalent malignant tumor threatening women's health, with approximately 1.4 million new cases and 400,000 deaths globally each year. HER2-positive breast cancer is characterized by higher malignancy, more rapid disease progression, a greater propensity for metastasis and recurrence, and a poorer prognosis; thus, it is more aggressive than other subtypes and is often referred to as the "most dangerous form of breast cancer." Although studies have demonstrated that trastuzumab therapy can reduce the risk of recurrence in early-stage HER2-positive breast cancer after surgery, 25% of patients treated with trastuzumab still experience recurrence.

NERLYNX is a kinase inhibitor indicated for extended adjuvant treatment of adult patients with HER2-positive breast cancer with brain metastases, following adjuvant trastuzumab-based therapy. The recommended dosage of NERLYNX is 240 mg (six 40 mg tablets) taken orally once daily for one year. The most common adverse reaction associated with NERLYNX is diarrhea; antidiarrheal prophylaxis should be initiated with the first dose and continued during the first two months (56 days) of treatment. To help ensure patient access to NERLYNX, Puma has implemented the Puma Patient Lynx support program, which provides reimbursement services and financial assistance to patients.

Puma Biotechnology, founded in 2010 and headquartered in Los Angeles, USA, is a biopharmaceutical company. The company focuses on the development and commercialization of innovative oncology drugs. Puma’s main products include PB272 (neratinib, oral), PB272 (neratinib, intravenous), and PB357. PB272 received FDA approval in July 2017 and is marketed in the United States under the brand name NERLYNX. PB272 received marketing authorization from the European Commission in September 2018.

Alan H. Auerbach, CEO and President of Puma Biotechnology, stated, “The FDA’s orphan drug designation marks the progress and commitment in our development of treatments for patients with HER2-positive breast cancer. Although there have been advances in treatment regimens for HER2-positive breast cancer, brain metastases in these patients remain a significant clinical challenge and contribute to the high morbidity and mortality observed in this population. The blood-tumor barrier penetration of NERLYNX provides a better therapeutic option for these patients.”

(Compiled by Hu Yifan)