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Supplemental NDA Accepted and Priority Review Granted for Bortezomib To Treat Second-Line Multiple Myeloma

CAMBRIDGE, Mass., Dec. 1 /PRNewswire-FirstCall/ -- Millennium Pharmaceuticals, Inc. today announced the U.S. Food and Drug Administration (FDA) accepted for review the Company's supplemental New Drug Application (sNDA) and granted Priority Review designation for VELCADE for the treatment of patients with multiple myeloma who have received at least one prior therapy. The submission was based on data from the phase III APEX study that compared VELCADE to a traditional myeloma therapy, high-dose dexamethasone, which demonstrated a statistically significant survival in patients receiving VELCADE. A patient population of approximately 22,000 in the U.S. could potentially benefit from a new second-line treatment option.

Priority Review of a sNDA is granted by the FDA for a treatment that addresses an unmet medical need. The FDA expedites the approval process for such a sNDA by reducing the standard review period for the application from ten months to six months. Acceptance of the filing means the FDA has made a threshold determination that the sNDA is sufficiently complete to permit a substantive review.

"We are excited that more patients may soon have access to VELCADE earlier in their treatment where VELCADE has shown a significant improvement over a standard therapy in delaying disease progression and improved survival," said David Schenkein, M.D., vice president, clinical oncology development, Millennium. "Millennium is committed to making a difference in patients' lives, and will continue in partnership with Johnson & Johnson Pharmaceutical Research and Development L.L.C., to extensively research the benefits of VELCADE extensively across the multiple myeloma treatment paradigms as well as in other hematologic and solid tumors."

The acceptance of this supplementary filing comes approximately eighteen months after the FDA approval of 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. 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.

APEX Results

The sNDA submission was based primarily upon the results of the phase III APEX confirmatory study that compared VELCADE to high-dose dexamethasone. The APEX trial was halted one year early after an independent data monitoring committee concluded the findings of a pre-specified interim analysis showed a statistically significant improvement in time-to-disease progression in favor of VELCADE.

The APEX trial enrolled 669 patients with relapsed or refractory multiple myeloma (patients had received one to three prior therapies) at 93 centers in North America, Europe and Israel. Preliminary results presented at ASCO 2004 included:

* A statistically significant survival advantage (p<.01 in="" patients="" treated="" the="" velcade="" alone="" arm.="" importantly="" statistical="" significance="" was="" maintained="" even="" though="" approximately="" percent="" of="" crossed="" over="" to="" receive="" after="" experiencing="" progressive="" disease="" on="" dexamethasone="" arm="" during="" first="" year="" there="" an="" estimated="" reduction="" risk="" death="" receiving="" compared="" those="" dexamethasone.="" data="" from="" interim="" analysis="" showed="" a="" improvement="" time="" progression="" months="" high-dose="" incidence="" adverse="" events="" similar="" between="" two="" groups.="" overall="" grade="" rates="" were="" and="">

The final APEX data, including updated survival data, response rates and time-to-progression data, will be presented at the American Society of Hematology (ASH) 46th annual meeting in San Diego, Calif. on December 6, 2004.

VELCADE is currently approved in the United States, 27 European Union states, Argentina, Israel, and South Korea. VELCADE is being co-developed by Millennium and Johnson & Johnson Pharmaceutical Research & Development, L.L.C. Millennium is responsible for commercialization of VELCADE in the U.S.; Ortho Biotech and Janssen-Cilag are responsible for commercialization in Europe and the rest of the world. Janssen Pharmaceutical K.K. is responsible for Japan.

About VELCADE® (bortezomib) for Injection

VELCADE is approved 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 improvement in survival. VELCADE is contraindicated in patients with hypersensitivity to bortezomib, boron, or mannitol.

Risks associated with VELCADE® (bortezomib) for Injection therapy include new or worsening peripheral neuropathy, orthostatic hypotension, congestive heart failure, gastrointestinal adverse events, thrombocytopenia, and tumor lysis syndrome. Women of childbearing potential should avoid becoming pregnant while being treated with VELCADE.

In 228 patients who were treated with VELCADE 1.3 mg/m2/dose in phase II studies, the most commonly reported adverse events were asthenic conditions (65%), nausea (64%), diarrhea (51%), decreased appetite including anorexia (43%), constipation (43%), thrombocytopenia (43%), peripheral neuropathy (37%), pyrexia (36%), vomiting (36%), and anemia (32%). Fourteen percent of patients experienced at least one episode of Grade 4 toxicity, with the most common toxicities being thrombocytopenia (3%) and neutropenia (3%). A total of 113 (50%) of the 228 patients experienced Serious Adverse Events (SAEs) during studies. The most commonly reported SAEs included pyrexia (7%), pneumonia (7%), diarrhea (6%), vomiting (5%), dehydration (5%), and nausea (4%).

For more information about VELCADE clinical trials, patients and physicians can contact the Millennium Medical Product Information Department at 1-(866)-VELCADE.

Source: Millennium Pharmaceuticals, Inc.

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