On May 13, 2003, the FDA approved bortezomib (Velcade®, a trademark of Millennium Pharmaceuticals, Inc.) for the treatment of multiple myeloma patients who have received at least two prior therapies and have demonstrated disease progression on the last therapy.

Bortezomib was evaluated in 256 patients with multiple myeloma in two open-label multicenter studies conducted in the United States. In the primary efficacy study, 202 patients were entered after having received at least two prior therapies and progressing on the most recent therapy. Bortezomib was administered intravenously at 1.3 milligrams (mg) /m2/dose twice weekly for 2 weeks, followed by a 10-day rest period (21 day treatment cycle) for a maximum of 8 treatment cycles. In the study population, the median number of prior therapies was six, and 64 percent of patients had received stem cell transplant or other high dose therapy. Results (Blade criteria) in the 188 eligible and evaluable patients included complete responses in 5 patients, for a complete response rate of 2.7 percent (95 CI: 1 percent, 6 percent); partial responses occurred in 47 patients for a PR rate of 25 percent (95 CI: 19 percent, 32 percent). Clinical remissions by SWOG criteria were observed in 17.6 percent of patients (95CI: 12 percent, 24 percent). Median duration of response was 365 days.

Adverse events (AEs) occurring in greater than 50 percent of patients included fatigue or malaise, nausea, and diarrhea. AEs occurring in greater than 30 percent of patients were anorexia, constipation, thrombocytopenia, peripheral neuropathy, pyrexia, vomiting, and anemia. Severe AEs with incidences greater than 10 percent were thrombocytopenia, peripheral neuropathy, neutropenia and asthenia.

Bortezomib approval is based on objective response rate and response duration under accelerated approval provisions. Randomized controlled clinical trials will be performed to evaluate whether bortezomib treatment is associated with clinical benefit. Bortezomib received fast-track designation and priority review. The filing date of this application was January 21, 2003.

Full prescribing information is available, including clinical trial information, safety, dosing, drug-drug interactions and contraindications.

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