Celgene's Amrubicin granted orphan drug designation by US FDA

Celgene Corporation's Amrubicin has been granted orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of small cell lung cancer.

FDA's Orphan Drug Act was designed to encourage the development of products that demonstrate promise for the diagnosis, prevention and/or treatment of life-threatening or very serious conditions that are rare and affect 200,000 persons or less in the United States. Orphan drug designation provides an important economic incentive for the development of new products in the cancer field. US orphan drug designation provides for seven years of market exclusivity, reduction in regulatory fees, and additional regulatory support for R&D initiatives.

"The decision by the FDA to grant Amrubicin orphan drug designation in this critical area of oncology advances our efforts to deliver innovative therapies to patients in areas of unmet medical need and represents the first such milestone in the area of solid tumour cancers," said Graham Burton M.D., SVP, Global Regulatory Affairs and Pharmacovigilance for Celgene Corporation. "We will continue to work diligently with regulators as we advance this promising next generation anthracycline through clinical development."

Amrubicin is a third-generation, synthetic anthracycline analogue that has demonstrated substantial clinical efficacy in the treatment of small cell lung cancer. Amrubicin is a potent topoisomerase II inhibitor and is being studied as a single agent and in combination with anti-cancer therapies for a variety of solid tumours, including lung and breast cancers.

Amrubicin is currently approved and marketed in Japan for the treatment of small cell lung cancer by Nippon Kayaku, a Japanese pharmaceutical firm focused on oncology, which licensed Japanese marketing rights from Dainippon Sumitomo, the original developer of the therapy. Dainippon Sumitomo also licensed the US and European rights of Amrubicin to Pharmion Corporation, which was acquired by Celgene Corporation in 2008.

Patients diagnosed with SCLC each year in the US and EU, approximately 60 percent of patients have extensive disease at diagnosis, and the remaining 40 percent present with localized, or limited stage, disease.