US biotech company Puma Biotechnology has secured orphan drug designation to NERLYNX (neratinib) from the US Food and Drug Administration (FDA) for the treatment of breast cancer patients having brain metastases, as per the latest pharma regulatory news.
The FDA orphan drug designation program is given to investigational drugs designed for the treatment, prevention, or diagnosis of rare medical diseases or conditions that impact lesser than 200,000 individuals in the US.
Orphan designation qualifies sponsors for various important benefits and incentives like opportunities for grant funding towards clinical trial costs, user fee waivers, tax credits, and the possibility for a seven-year period of marketing exclusivity after FDA approval of the investigational drug for the indication for which it was granted orphan designation.
Commenting on the NERLYNX FDA orphan drug designation, Alan H. Auerbach – Chairman, CEO and President of Puma Biotechnology said: “Receiving Orphan Drug Designation from the FDA signifies our continued progress and commitment to developing treatments for patients with HER2-positive breast cancer.
“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.”
NERLYNX is a kinase inhibitor approved by the FDA for the extended adjuvant treatment of adult patients having HER2 overexpressed/amplified breast cancer, after adjuvant trastuzumab-based therapy.
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