Nerlynx Granted FDA’s Orphan Drug Status for Treating Breast Cancer with Brain Metastases

Nerlynx Granted FDA’s Orphan Drug Status for Treating Breast Cancer with Brain Metastases
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Puma Biotechnology’s Nerlynx (neratinib) for the treatment of HER2-positive breast cancer in women with brain metastases. Between 10 to 15% of people with metastatic breast cancer develop brain metastases. However, that number reaches up to 30% in women with advanced HER2-positive breast cancer. Orphan drug designation provides Puma with benefits, including financial incentives for therapy development and commercialization. It also provides U.S. market exclusivity for a period of seven years following the medication's approval, FDA support for clinical studies, and special fee exemptions and reductions. The designation is given to investigative treatments aimed at helping people with a rare disease, which means it affects fewer than 200,000 people in the U.S. “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. Nerlynx was approved by the FDA in July 2017 as an extended, adjuvant therapy — an added treatment to lower the risk that the cancer will come back — for adults with early-stage HER2-positive breast cancer. The treatment was designed to follow adjuvant therapy with trastuzumab (brand names Herceptin, and others). The active substance, neratinib, is a small molecule that works as an irreversible tyrosine kinase inhibitor (TKI), blocking the epidermal growth factor receptor HER2, as well as other receptors. It is designed to inhibit HER2 — overexpressed by some breast tumors and associated with a more aggressive disease — to prevent cancer cell proliferat
Subscribe or to access all post and page content.