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Accelerated FDA approval of Velcade for multiple myeloma treatment

ROCKVILLE, MD., May 13, 2003 -- The FDA announced approval of Velcade (bortezomib) injection, a new treatment for multiple myeloma, a cancer of the bone marrow.

FDA reviewed the application for this drug in less than four months. Velcade, distributed and marketed by Millennium Pharmaceuticals, is the first in a new class of anticancer agents known as proteasome inhibitors.

Velcade was approved under the accelerated approval program. The program helps make promising products for serious or life threatening diseases available earlier in the development process by allowing approval to be based on a promising effect of the drug, such as tumor shrinkage, before there is actual evidence of improved survival or other clinical benefit. The drug's commercial sponsor worked closely with FDA to define the studies that would be conducted.

"As a result of close collaboration among the company, The National Cancer Institute, and FDA in the development and review of the drug, FDA was able to make this novel therapy available sooner to help many thousands of patients suffering from multiple myeloma." said FDA Commissioner Mark B. McClellan, M.D., Ph.D. "The approval of Velcade illustrates FDA's commitment to providing patients with access to safe and effective drugs as quickly as possible."

Velcade is indicated for patients whose disease has relapsed after two prior treatments and who have demonstrated resistance to their last treatment.

FDA evaluated the safety and efficacy of Velcade based on a study of 202 patients who had received at least two prior therapies and demonstrated disease progression on their most recent therapy. Altogether, out of 188 patients evaluated for response, twenty eight percent showed a response to Velcade. The response lasted a median time of one year. Another trial in 54 patients with relapsed multiple myeloma showed similar responses.

As yet there are no controlled trials of Velcade demonstrating clinical benefit, such as improvement in survival. To address this issue, Velcade's developer will perform additional studies after approval. These will include the completion of an on-going study and an additional study comparing Velcade to standard therapy.

"The drug shows a significant effect on patients with multiple myeloma that have not responded to other treatments a response that is likely to result in significant clinical benefit," said Dr. McClellan. "As with other treatments approved under our accelerated approval process, further studies are necessary for clarifying Velcade's clinical benefits."

The most commonly reported adverse events reported in clinical trials include nausea, fatigue, diarrhea, constipation, headache, decreased appetite, decreased platelets and red cells in the blood, fever, vomiting, and peripheral neuropathy (numbness and tingling, and occasionally pain in the extremities).

Multiple myeloma is the second most prevalent blood cancer after non-Hodgkin's lymphoma. It is a cancer of the plasma cell, an important part of the immune system that produces antibodies to help fight infection and disease. There are approximately 45,000 people in the United States living with multiple myeloma and an estimated 14,600 new cases of multiple myeloma are diagnosed each year.

Source: FDA

Posted: May 2003