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FDA Approves Velcade

FDA Approves Velcade (bortezomib) for Injection for the Treatment of Relapsed and Refractory Multiple Myeloma

CAMBRIDGE, Mass., May 13, 2003 /PRNewswire-FirstCall/-- Millennium Pharmaceuticals, Inc. (Nasdaq: MLNM) today received approval from the U.S. Food and Drug Administration (FDA) to market Velcade for the treatment of multiple myeloma patients who have received at least two prior therapies and have demonstrated disease progression on the last therapy. The effectiveness of Velcade is based on response rates. There are no controlled trials demonstrating a clinical benefit such as an improvement in survival. Velcade, the first of a new class of medicines called proteasome inhibitors, is the first treatment in more than a decade to be approved for patients with multiple myeloma - a cancer of the blood.

"The FDA approval of Velcade represents a major advance in our fight against multiple myeloma," said Kenneth Anderson, M.D., from Dana-Farber Cancer Institute in Boston, Mass. "With its new and unique mechanism of action of inhibiting the proteasome, Velcade is different from traditional chemotherapies and represents a new treatment option for patients."

"Millennium was established with the goal of using innovative science to develop novel products that would address the unmet medical needs of patients," said Mark Levin, chief executive officer and chairperson of Millennium. "Our success in bringing Velcade to patients so rapidly reflects the high level of collaboration among many partners, both internally and externally. Moving forward, Millennium will continue its mission of developing breakthrough products that make a difference in patients' lives."

Velcade and proteasome inhibition represent a completely new approach to treating multiple myeloma. The development of this product is based on the Company's deep understanding of cancer disease pathways and the effect of proteasome inhibition on those pathways. The proteasome is an enzyme complex that exists in all cells and plays an important role in degrading proteins that control the cell cycle and cellular processes. By blocking the proteasome, Velcade disrupts numerous biologic pathways, including those related to the growth and survival of cancer cells.

Priority Review

Velcade(TM) (bortezomib) for Injection was granted Priority Review by the FDA on March 10, 2003 and was approved approximately two months later. In addition, it has been just four and a half years from the first human dose to FDA approval, an indication of the importance of Velcade and proteasome inhibition. As a result of the accelerated approval process and the close collaboration between Millennium, the FDA, the National Cancer Institute and other academic and medical institutions, the development and approval of Velcade is among the most rapid for a cancer treatment. As part of this accelerated approval, Millennium will be completing preclinical and phase IV clinical studies, including a study of previously untreated multiple myeloma patients.

"Today, the thousands of people living with multiple myeloma in the United States have been given a new treatment option," said Kathy Giusti, president, Multiple Myeloma Research Foundation (MMRF), and a myeloma patient. "The MMRF has been committed to helping accelerate the search for a cure for multiple myeloma and we are proud to have been able to work with Millennium in bringing this important new therapy to patients."

"Velcade is the kind of cutting-edge treatment for which we have been advocating, and provides a new treatment option for the thousands of patients with this disease," said Susie Novis, president, International Myeloma Foundation (IMF). "The approval of Velcade represents a major milestone in our quest to see new treatments made available to patients with multiple myeloma."

Clinical Results

The FDA approval of Velcade is based primarily upon the results of a major multicenter phase II open-label, single-arm (SUMMIT) trial, which included 202 patients with relapsed and refractory multiple myeloma receiving at least two prior therapies (median of six). Patients had advanced disease, with 91 percent refractory to their most recent therapy prior to study entry. Response rates were independent of the number or type of previous therapies.

Key findings for the 188 patients evaluable for response showed:
-- Overall, the response rate for complete and partial responders was 27.7
percent (criteria per Blade, et al(1)) (95 percent CI; 21, 35);
-- Significantly, 17.6 percent - or almost one out of every five patients
- experienced a clinical remission(2) (SWOG criteria) (95 percent CI;
12, 24);
-- The median survival for all patients was 16 months (range was less than
one to greater than 18 months);
-- The median duration of response for complete and partial responders was
12 months (95 percent CI; 224 days; NE); and
-- Side effects were generally predictable and manageable.

Important Safety Information About Velcade

Velcade(TM) (bortezomib) for Injection is contraindicated in patients with hypersensitivity to bortezomib, boron, or mannitol.

In 228 patients who were treated with Velcade in two phase II studies of multiple myeloma, the most commonly reported adverse events were asthenic conditions (including fatigue, malaise and weakness) (65 percent), nausea (64 percent), diarrhea (51 percent), appetite decreased (including anorexia) (43 percent), constipation (43 percent), thrombocytopenia (43 percent), peripheral neuropathy (including peripheral sensory neuropathy and peripheral neuropathy aggravated) (37 percent), pyrexia (36 percent), vomiting (36 percent), and anemia (32 percent). Fourteen percent of patients experienced at least one episode of grade four toxicity, with the most common toxicity being thrombocytopenia (three percent) and neutropenia (three percent).

About Multiple Myeloma

Multiple myeloma (also known as myeloma or plasma cell myeloma) is a cancer of the blood in which malignant plasma cells are overproduced in the bone marrow. Plasma cells are white blood cells that help produce antibodies called immunoglobulins that fight infection and disease. However, most patients with multiple myeloma have cells that produce a form of immunoglobulin called paraprotein (or M protein) that does not benefit the body. In addition, the malignant plasma cells replace normal plasma cells and other white blood cells important to the immune system. Multiple myeloma cells can also attach to other tissues of the body, such as bone, and produce tumors. The cause of the disease is unknown.

Multiple myeloma is the second most common cancer of the blood, representing approximately one percent of all cancers and two percent of all cancer deaths. It is estimated that approximately 45,000 Americans have multiple myeloma with about 15,000 new cases diagnosed each year. Only about 30 percent of multiple myeloma patients survive longer than five years with the disease. Although the disease is predominantly a cancer of the elderly (the average age at diagnosis is 70 years of age) recent statistics indicate both increased incidence and younger age of onset.

Ongoing Clinical Trials

Millennium is conducting an international, multicenter phase III APEX trial of Velcade in patients with either relapsed or refractory multiple myeloma as well as two phase II trials with Velcade, one in patients with metastatic colorectal cancer and another in patients with advanced non-small cell lung cancer. In addition, the Company also has several ongoing phase I and II trials in patients with various hematologic and solid tumors and plans to initiate additional phase I and II studies later this year. For more information about Velcade clinical trials, patients and physicians can contact the Medical Information Line at (866) Velcade.

About Millennium

Millennium Pharmaceuticals, Inc., a leading biopharmaceutical company, co- promotes INTEGRILIN(R) (eptifibatide) Injection, a market-leading cardiovascular product, and has a robust clinical development pipeline of product candidates. The Company's research, development and commercialization activities are focused in four areas: cardiovascular disease, oncology and inflammatory and metabolic diseases. By applying its knowledge of the human genome, its understanding of disease mechanisms, and its industrialized technology platform, the Company is developing breakthrough personalized medicine products. Headquartered in Cambridge, Mass., the Company also has facilities in South San Francisco, Calif. and Cambridge, UK.

This press release contains "forward-looking statements," including statements about our growth and future operating results, discovery and development of products, potential acquisitions, strategic alliances and intellectual property. Various risks may cause the Company's actual results to differ materially, including: adverse results in our drug discovery and clinical development processes; failure to obtain patent protection for our discoveries; commercial limitations imposed by patents owned or controlled by third parties; our dependence upon strategic alliance partners to develop and commercialize products and services based on our work; difficulties or delays in obtaining regulatory approvals to market products and services resulting from our development efforts; the commercial success of INTEGRILIN(R) (eptifibatide) Injection and Velcade(TM) (bortezomib) for Injection; and the requirement for substantial funding to conduct research and development and to expand commercialization activities. For a further list and description of the risks and uncertainties we face, see the reports we have filed with the Securities and Exchange Commission. We disclaim any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

(1) The criteria defined by Blade, et al are an assessment standard used
to describe changes in disease status, including a confirmation 6
weeks later. The criteria incorporate a full clinical analysis of
disease response, specifically, a "complete response" required 100
percent disappearance of the original myeloma protein from the blood
and urine on at least two determinations at least six weeks apart by
immunofixation; less than five percent plasma cells in the bone
marrow; no increase in size or number of lytic bone lesions; and
disappearance of soft tissue tumors (plasmacytomas). "Partial
response" required >/=50 percent reduction in serum myeloma protein
and >/=90 percent reduction of urine myeloma protein on at least two
occasions for a minimum of six weeks, stable bone disease and calcium

(2) SWOG clinical remission criteria were a 75 percent reduction in serum
myeloma protein and/or a 90 percent reduction in urine protein on at
least two occasions for a minimum of at least six weeks, stable bone
disease and calcium.

SOURCE Millennium Pharmaceuticals, Inc.

Posted: May 2003

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