Cyclacel Pharmaceuticals, Inc, a biopharmaceutical company developing oral therapies that target the various phases of cell cycle control for the treatment of cancer and other serious disorders, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to the company’s sapacitabine (CYC682) product candidate for the treatment of both acute myeloid leukaemia (AML) and myelodysplastic syndromes (MDS).

“Orphan drug designation for both AML and MDS significantly strengthens the value proposition represented by sapacitabine and enhances our opportunity to advance this promising product candidate to late stage clinical development and commercialization,” said Spiro Rombotis, president and chief executive officer of Cyclacel.

Sapacitabine, a cell cycle modulating nucleoside analogue, is in phase-2 studies for the treatment of AML in the elderly, MDS and lung cancer. Cyclacel has reported phase-2 results from ongoing studies in AML and MDS. The company plans to advance sapacitabine into pivotal phase-3 development in 2010. During the first quarter of 2010, the company submitted a Special Protocol Assessment (SPA) request to the US FDA for a randomized phase-3 study of sapacitabine in elderly patients with AML.

Orphan drug designation entitles Cyclacel Pharmaceuticals to seven years of marketing exclusivity for sapacitabine upon regulatory approval, as well as the opportunity to apply for grant funding from the US government to defray costs of clinical trial expenses, tax credits for clinical research expenses and a potential waiver of the US FDA's application user fee. Orphan status is granted by the US FDA to promote the development of new drug therapies for the treatment of diseases that affect fewer than 200,000 individuals in the United States.

Cyclacel is a biopharmaceutical company developing oral therapies that target the various phases of cell cycle control for the treatment of cancer and other serious disorders.