Thalidomide Receives Positive Opinion For Multiple Myeloma Treatment
European Medicines Agency (EMEA) has issued a positive opinion to recommend approval of Pharmion Corporation's Thalidomide Pharmion for use in combination with melphalan and prednisone as first line treatment for patients with untreated multiple myeloma (MM), aged 65 years or older or ineligible for high dose chemotherapy. The marketing authorization application (MAA) for Thalidomide Pharmion was submitted to the EMEA in January 2007.
The EMEA's Committee for Medicinal Products for Human Use (CHMP) reviewed the application, and its positive opinion will be forwarded to the European Commission (EC), which generally follows, but is not obligated to follow, the recommendation of the CHMP, and issues final marketing approval within two to three months. Once ratified by the EC, a single marketing authorization would be granted to Pharmion to market Thalidomide Pharmion for first line multiple myeloma in the 27 member states of the European Union (EU) as well as Norway and Iceland.
"We are very pleased with this recommendation by the EMEA for the approval of Thalidomide in the treatment of first line multiple myeloma, and clearly appreciate that the return of Thalidomide to Europe as an approved therapy represents a significant step toward the safe distribution of this important and widely used drug for patients with multiple myeloma," said Patrick J. Mahaffy, Pharmion's president and chief executive officer. "This has been a long and complex process and I would like to thank all of the participants in the dialogue that has led to this recommendation by the EMEA, which include European thalidomide groups, patient organizations, the hematology community and the employees of Pharmion who made this happen. We have been and will remain committed to managing Thalidomide with the respect that its history requires."
The Marketing Authorization Application reviewed by the EMEA is based upon a clinical data package comprised of studies of nearly 1400 patients in total, including the Intergroupe Francophone du Myelome (IFM) 99-06 survival study. The three-arm study conducted by IFM demonstrated the superiority of melphalan/prednisone plus Thalidomide (MPT) over standard therapy of melphalan/prednisone (MP) or a combination of chemotherapies (vincristine/adriamycin/dexamethasone (VAD)) followed by melphalan and transplantation (MEL 100) in the treatment of newly diagnosed multiple myeloma patients, aged 65 to 75 who were ineligible for intensive bone marrow transplantation.
A total of 447 patients were randomized to one of the three treatment arms. At final analysis, the overall median survival in the MPT arm was 51.6 months, compared to 33.2 and 38.3 months, respectively, for the MP and MEL 100 arms. The hazard ratios were 0.59 and 0.69, respectively. Thalidomide treatment was generally well-tolerated by the majority of patients. The most frequently reported adverse events associated with MPT included neutropenia, somnolence and constipation. The Thalidomide combination was also associated with a 5-10% greater risk of Grade 3 and 4 venous thromboembolism and peripheral neuropathy.
"Our study results demonstrate the profound impact on overall survival that the addition of Thalidomide to the standard of care can provide to patients with untreated multiple myeloma," said Professor Philippe Moreau, Professor of Clinical Hematology at Nantes Faculty of Medicine and Chairman of the Intergroupe Francophone du Myelome. "This recommendation recognizes the importance of the study results, opens drug access to all physicians and patients in Europe and provides great hope for patients with this devastating disease."
Multiple myeloma, the second most common cancer of the blood, affects approximately 82,000 people in the EU, and approximately 25,000 people in the EU are diagnosed with multiple myeloma each year.