PRINCETON, N.J.--(BUSINESS WIRE)-- Celator Pharmaceuticals today announced that the European Commission (EC) has granted orphan drug status to CPX-351, the company’s proprietary “liposomal combination of cytarabine and daunorubicin,” for the treatment of acute myeloid leukemia (AML). The designation follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA).
Orphan status is granted to products that can be used in the diagnosis, treatment, or prevention of life-threatening or debilitating diseases with incidences of no more than 5 in 10,000 in the European Union. In order to receive orphan status, there must also be either no satisfactory method of diagnosis, prevention or treatment of the authorized condition, or, if such a method does exist, the medicine must be of significant benefit to those affected by the condition. Orphan status confers 10 years of marketing exclusivity in all EU member countries following approval and a range of other benefits during the development and regulatory review process including scientific assistance for study protocols, access to a centralized review process covering all member countries, and reduction or elimination of registration and market authorization fees.
“We are very pleased that the EMA has recognized the potential of CPX-351 to substantially improve patient outcomes in the treatment of AML in Europe,” said Scott Jackson, chief executive officer of Celator Pharmaceuticals. “Receiving orphan status is an important achievement in our strategy to develop CPX-351 in this patient population which urgently needs therapeutic advances.”
Celator previously announced that the U.S. Food & Drug Administration (FDA) granted orphan drug designation to CPX-351 (Cytarabine:Daunorubicin) Liposome Injection for the treatment of acute AML.
CPX-351 (Cytarabine:Daunorubicin) Liposome Injection represents a new approach to developing combinations of drugs in which drug molar ratios with synergistic anti-tumor activity are encapsulated in a drug delivery vehicle in order to maintain the desired ratio following administration. CPX-351 has been granted orphan drug status by the U.S. Food & Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML). CPX-351 is currently in phase 2 clinical development for the treatment of AML. Celator has completed two successful randomized, phase 2 studies. The first study compared CPX-351 to the standard "7+3" regimen of cytarabine:daunorubicin in patients 60-75 years of age with newly diagnosed AML and the second study compared CPX-351 versus intensive salvage therapy in first relapse AML patients, age 18-65 years. The second study is supported by The Leukemia & Lymphoma Society®. Celator is preparing CPX-351 for Phase 3 clinical development.
About Celator Pharmaceuticals, Inc.
Celator Pharmaceuticals, Inc., with locations in Princeton, NJ, and Vancouver, BC, is a privately held pharmaceutical company developing new and more effective therapies to treat cancer. CombiPlex®, the company's proprietary drug ratio technology platform, represents a novel approach that identifies molar ratios of drugs that will deliver a synergistic benefit, and locks the desired ratio in a nano-scale drug delivery vehicle that maintains the ratio in patients with the goal of improving clinical outcomes. The company pipeline includes two Phase 2 products; CPX-351 (a liposomal formulation of cytarabine:daunorubicin) for the treatment of acute myeloid leukemia and CPX-1 (a liposomal formulation of irinotecan:floxuridine) for the treatment of colorectal cancer; a preclinical stage compound, CPX-571 (a liposomal formulation of irinotecan:cisplatin); and multiple research programs, including the hydrophobic docetaxel prodrug nanoparticle (HDPN) formulation being studied by the National Cancer Institute's Nanotechnology Characterization Laboratory. Based on the applications of CombiPlex and the proprietary nanoparticle prodrug delivery platform, Celator is positioned to advance a broad pipeline of cancer therapies involving both previously approved and novel drug agents. For more information, please visit the company's website at www.celatorpharma.com. Information on ongoing trials is available at www.clinicaltrials.gov.
KEYWORDS: United States Europe North America New Jersey
INDUSTRY KEYWORDS: Health Biotechnology Oncology Pharmaceutical FDA