Celgene Corporation, an independent biopharmaceutical company, announced that Revimid (CC-5013) received fast track designation from the U.S. Food and Drug Administration (FDA) for the treatment of relapsed or refractory multiple myeloma. The FDA grants fast track designation to compounds that may provide a significant improvement in the safety or effectiveness of the treatment for a serious or life-threatening disease. Compounds that receive the designation are also eligible for priority review, which provides for a six-month review of a new drug application by the FDA.

"We are pleased that the FDA granted Revimid fast track status in relapsed or refractory multiple myeloma," said Sol J. Barer, Ph.D., president and chief operating officer of Celgene Corporation. "We are very encouraged by the recently published and presented data on Revimid, and based on the data, we are moving Revimid through the clinical and regulatory development process as quickly as possible to help alleviate the unmet medical needs of late-stage cancer patients."

Celgene is currently evaluating Revimid as a potential treatment for relapsed or refractory multiple myeloma in a multi-center Phase II trial led by investigators at the Dana-Farber Cancer Institute at Harvard University. At the annual meeting of the American Society of Hematology in December, Dr. Paul Richardson, one of the trial's lead investigators, presented preliminary results from the trial. He reported that 39 of 46 evaluable patients (85 per cent) with progressive disease experienced a reduction or stabilization in their paraprotein levels while on Revimid monotherapy. Importantly, five of seven patients who experienced progressive disease on Revimid monotherapy subsequently experienced a reduction or stabilization of paraprotein levels after Revimid plus dexamethasone combination therapy. Therefore, 44 of 46 evaluable patients (96 per cent) experienced a reduction or stabilization of paraprotein levels after either Revimid monotherapy or Revimid plus dexamethasone combination therapy.

During the trial some patients experienced grade three or four myelosuppression and grade one or two diarrhea, fever, muscle cramps, neuropathy, constipation, rash and fatigue.

Celgene recently initiated pivotal programs for Revimid in relapsed and refractory multiple myeloma and metastatic melanoma. Each pivotal program consists of multi-center, controlled, double-blind trials that are being conducted in the United States and internationally. Celgene finalized both programs after the FDA reviewed the protocols through the Special Protocol Assessment Program.

IMiDs are novel, small molecule, orally available compounds that modulate the immune system. Revimid is one of many compounds in the IMiD pipeline. In addition to multiple myeloma, Revimid is also being evaluated in myelodysplastic syndromes, metastatic melanoma, glioblastoma and serious inflammatory diseases. Actimid (CC-4047), the next IMiD, is being tested in a Phase I/II clinical trial in refractory multiple myeloma. Interim data from this trial was presented at the annual meeting of the International Society for Experimental Hematology in early July and demonstrated that Actimid has anti-tumor activity in multiple myeloma and has an acceptable toxicity profile. Actimid and Revimid have different activity profiles that may be better suited for different disease types. A third IMiD is currently in preclinical evaluation. The IMiD pipeline is covered by a comprehensive intellectual property estate of U.S. and foreign issued patents and pending patent applications including composition-of-matter and use patents.

There are approximately 50,000 people in the United States living with multiple myeloma, and about 14,600 new cases of multiple myeloma are diagnosed each year, making it the second most common blood cancer. Multiple myeloma is a malignant cancer of the plasma cell, which is a type of white blood cell found in the bone marrow. As the cancer grows it destroys normal bone tissue, causing pain and crowding out normal cell production. About 11,000 Americans are expected to die of multiple myeloma in 2003.

Revimid (CC-5013) and Actimid (CC-4047) are not approved by the FDA and are currently being evaluated in clinical trials for efficacy and safety for future regulatory applications.