ChemGenex's Marketing Authorization Application For Omacetaxine Mepesuccinate Validated By The European Medicines Agency

Main Category: Lymphoma / Leukemia / Myeloma
Also Included In: Regulatory Affairs / Drug Approvals;  Cancer / Oncology;  Blood / Hematology
Article Date: 30 Nov 2009 - 4:00 PST

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ChemGenex Pharmaceuticals Limited (ASX:CXS) announced that the European Medicines Agency ("EMEA") has determined that the Marketing Authorization Application ("MAA") for omacetaxine mepesuccinate for the treatment of chronic myeloid leukemia (CML) patients who have failed treatment with imatinib and who have developed the Bcr-Abl T315I mutation is valid. Validation of the MAA indicates that ChemGenex's application, submitted on 29 October 2009, is complete and that the review process has begun. The filing is based on data from pivotal Study 202 where omacetaxine demonstrated clinical benefit for the treatment of T315I positive CML patients.

This additional regulatory milestone follows the acceptance earlier this month of a New Drug Application (NDA) by the U.S. Food and Drug Administration (FDA) for marketing approval for omacetaxine (filed under the trade name Omapro™) in the U.S.A.

"The submission and validation of this MAA shortly after our NDA submission and acceptance shows the commitment by ChemGenex to offer a treatment option to patients suffering from this life threatening condition," said Greg Collier, PhD, Chief Executive Officer and Managing Director. "This milestone marks another major accomplishment for ChemGenex this year, and we remain dedicated to expanding the use of omacetaxine within CML as well as with other leukemias."

Omacetaxine has received Orphan Drug designation from the EMEA and the FDA, and has received fast track status and Priority Review from the FDA. The MAA filing has been submitted in accordance with the centralized procedure and will be assessed by the EMEA. An opinion by the EMEA is expected in the fourth quarter of 2010. If approved, the marketing license will be valid simultaneously in all EU Member States plus Iceland, Liechtenstein and Norway. Orphan Drug designation will give omacetaxine market exclusivity for a period of ten years.

About omacetaxine mepesuccinate

Omacetaxine mepesuccinate is administered subcutaneously and acts differently from TKIs. It may have a therapeutic advantage for patients who have failed TKIs. Omacetaxine is currently in global phase 2/3 clinical trials for CML and has been granted Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMEA) as well as Fast Track status by the FDA.

Omacetaxine is a first-in-class cetaxine with demonstrated clinical activity as a single agent in a range of hematological malignancies. Omacetaxine has a novel mechanism of action, specifically binding to the ribosomal A-site cleft and inhibiting protein translation of short-lived oncoproteins that are upregulated in leukemic cells (particularly Cyclin-D1, Mcl-1 and c-Myc). In addition, pre-clinical research presented at the 14th Congress of the European Hematology Association (EHA) in Berlin, Germany this summer, demonstrated that omacetaxine kills human CML stem cells that are known to be insensitive to TKIs.

About Chronic Myeloid Leukemia (CML) and the Bcr-Abl T315I Mutation

Chronic myeloid leukemia (CML) is a cancer of the bone marrow with a worldwide prevalence of approximately 200,000 patients. The bone marrow is responsible for the production of specialized cells that constitute blood; these cells include red blood cells (to carry oxygen around the body), thrombocytes (to help stop bleeding) and certain white cells (part of the body's defense system against infection). In patients with CML the cell production system is diseased and defective. Cells multiply uncontrollably and do not fully develop (differentiate) into functional blood cells.

The majority of CML patients initially respond well to treatments with drugs called tyrosine kinase inhibitors (TKIs). However, a significant proportion of patients fail, or become intolerant to, one or more TKIs. In many of these situations the cause of failure can be traced to the emergence of Bcr-Abl mutations. A common mutation called T315I renders CML resistant to all currently approved TKIs, and has created a significant unmet medical need in the management of CML.

Source
ChemGenex Pharmaceuticals Limited