Thalidomide approval for Celgene

Celgene Corporation, from Warren, NJ, has said that the Australian Drug Evaluation Committee has recommended for approval Pharmion's thalidomide for the treatment of both patients with relapsed and refractory multiple myeloma (MM) and for the treatment of cutaneous manifestations of erythema nodosum leprosum (ENL), a severe and debilitating condition associated with leprosy.

The milestone decision in Australia will represent the first approval worldwide for thalidomide as a therapy for relapsed and refractory multiple myeloma. The FDA approved thalidomide for the treatment of cutaneous manifestations of ENL in July 1998.

On November 19, 2001, Celgene granted Pharmion exclusive licenses for Europe and certain other territories outside the US to its intellectual property covering thalidomide and STEPS (System for Thalidomide Education and Prescribing Safety) as well as preclinical and clinical data necessary to support international regulatory filings for thalidomide. The STEPS system was developed in cooperation with the FDA as a method of using thalidomide by administering thalidomide while avoiding exposure to a developing fetus which includes restrictive distribution program for the prescribing and dispensing of Thalomid (Thalidomide).

Celgene presently holds a minority investment in Pharmion, as well as an exclusive option to purchase Penn T, the branch of Penn Pharmaceutical Services that manufactures thalidomide. This option, if exercised, would enable Celgene to receive both a 36% (less cost of goods sold) on their thalidomide sales and to manage the manufacturing of thalidomide as a wholly owned subsidiary of Celgene. Furthermore, Celgene has retained the rights for thalidomide in North America, China, Japan, Korea, and Taiwan.

Dr Sol Barer, president and coo at Celgene commented: 'This will be the first approval for a drug in multiple myeloma in Australia in many years. The approval of thalidomide in Australia will offer a meaningful option for myeloma patients and their families. Australia will be the first country to approve thalidomide for this deadly disease, and we look forward to similar outcomes in a number of other countries, where pivotal clinical trials are underway.'

The recommendation for approval of thalidomide in Australia reflects a real recognition of the critical need for new treatment options for multiple myeloma (MM). Multiple myeloma is the second most common cancer of the blood, representing approximately 1% of all cancers and 2% of all cancer deaths with a worldwide prevalence of approximately 200,000 cases. In the year 2002, there were an estimated 74,000 new cases of multiple myeloma worldwide. The estimated number of deaths from multiple myeloma in 2002 was 57,370 worldwide.