Shares in Celgene were given a boost yesterday after the US Food and Drug Administration agreed to assign orphan status to Amrubicin for the treatment of small cell lung cancer.

Amrubicin is a third-generation, synthetic anthracycline analogue that, according to Celgene, has shown “substantial clinical efficacy” in treating small cell lung cancer, a disease occurring almost exclusively in smokers. But the drug - a potent topoisomerase II inhibitor – is also currently being studied as a single agent and in combination with other anticancer therapies in variety of solid tumours, including lung and breast cancers, the group noted.

Shares in Celgene closed up nearly 2% at $61.34 on news of Amrubicin’s orphan drug status, which is granted to candidate therapies showing promise for the prevention, management or treatment of conditions affecting less than 200,000 patients in the US, and is designed to offer an incentive for companies to develop small-market drugs by providing seven years’ market exclusivity and regulatory fee cuts, for example.

“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,” commented Graham Burton, senior vice president of Global Regulatory Affairs and Pharmacovigilance at Celgene, and he stressed that the firm will “continue to work diligently with regulators” to advance “this promising next generation anthracycline through clinical development”.

Amrubicin is already available in Japan for the treatment of small cell lung cancer, where it is sold by Nippon Kayaku. European rights to the drug were originally licensed to Pharmion, which was acquired by Celgene for $2.9 billion earlier this year.