Celator Pharmaceuticals announced that the US Food & Drug Administration (FDA) has granted orphan drug designation to CPX-351 (Cytarabine: Daunorubicin) Liposome injection for the treatment of Acute Myeloid Leukaemia (AML).
Celator is currently preparing to conduct two randomized phase-2 studies with CPX-351. The first phase-2 study, in newly diagnosed, elderly patients with AML, is expected to start enrolling patients before the end of 2008. The second study, in AML patients who have relapsed following initial treatment, is projected to start enrolling patients in the first quarter of 2009.
Interim phase 1 data with CPX-351, where complete remissions were obtained in patients with advanced leukaemia, were reported in December 2007. The company submitted additional CPX-351 preclinical and clinical data to this year's American Society of Haematology (ASH) meeting in December and will report phase-1 results at that time.
CPX-351 is a liposomal formulation of cytarabine and daunorubicin delivered in a 5:1 molar ratio shown to be strongly synergistic in preclinical studies. CPX-351 was developed using Celator's proprietary CombiPlex technology platform.
Orphan drug status is granted to treatments for diseases that affect fewer than 200,000 people in the United States and provides the benefits of market exclusivity for seven years, tax credits, and a waiver of FDA user fees.
"The decision by FDA to grant CPX-351 orphan drug designation reinforces the importance of developing novel products for the treatment of rare diseases and represents another milestone for the company," said Scott Jackson, chief executive officer of Celator Pharmaceuticals. "We are committed to developing products that will benefit patients and look forward to initiating enrolment in our CPX-351 phase-2 programme."
Celator Pharmaceuticals, Inc, with locations in Princeton, New Jersey, and Vancouver, BC, is a privately held pharmaceutical company developing new and more effective therapies to treat cancer.