Bringing a new treatment option for patients with the fast-killing blood cancer multiple myeloma, Millennium Pharmaceuticals Inc. won FDA approval of the proteasome inhibitor Velcade (bortezomib) and plans to launch it next week.
The news, two months ahead of the FDA's deadline under the Prescription Drug User Fee Act, sent Millennium's stock (NASDAQ:MLNM) climbing up $1.90 Wednesday, or 15.2 percent, to close at $14.39.
"It was a great collaborative effort," said Kathy Giusti, president of the Multiple Myeloma Research Foundation (MMRF).
Velcade's approval comes only four and a half years from the first human dose, thanks to help from the MMRF, which provided funding and other guidance; the National Cancer Institute; the Dana-Farber Cancer Institute; and other academic and medical institutions.
The company filed for marketing clearance in January on evidence from a 202-subject Phase II study known as the SUMMIT trial. Data disclosed in December showed that Velcade increased median survival time in relapsed and refractory multiple myeloma patients to 16.4 months. They usually die six to nine months after they are diagnosed. (See BioWorld Today, Jan. 23, 2003, and Dec. 6, 2002.)
David Schenkein, vice president of oncology for Cambridge, Mass.-based Millennium, said the company has "over 40 clinical trials ongoing now in a wide range of hematologic and solid tumors," including one large randomized trial exploring the drug's efficacy in earlier forms of multiple myeloma.
The drug's wholesale price is $912 per vial, or about $20,000 per course of treatment - higher than many analysts expected, but Schenkein said Velcade "is priced very similar to other novel, first-in-class types of intravenous agents."
Millennium's revenue opportunity with Velcade in the U.S. market is about $200 million per year, according to estimates by MEDACorp, a division of Leerink Swann & Co.
In line with that, the company believes about one-fourth of the 45,000 myeloma patients in the U.S. have relapsed/refractory disease, which means a market of more than 11,000 patients, who will be targeted by the company's 75-member sales force.
A marketing deal is pending, Millennium officials said.
The proteasome is an enzyme complex involved in degrading proteins that control cellular processes. Velcade, by blocking the proteasome, is designed to disrupt pathways related to cancer cell growth.
As part of the accelerated approval, Millennium will complete preclinical and Phase IV clinical trials. Included is a study of previously untreated multiple myeloma patients.
"We haven't committed to a date for that yet," Schenkein said. More data regarding Velcade is expected at the American Society of Clinical Oncology meeting at the end of this month. Some will be preliminary data and some will be follow-up data from the SUMMIT trial.
"This mechanism of action may allow exploration in other areas," he added, although oncology is the focus now.
The MEDACorp poll involved 31 hematologists and oncologists treating 806 patients of the estimated 45,000 in the U.S., and might provide a clue to the potential adoption rate of Velcade.
Surveyors asked about Millennium's drug as compared to Warren, N.J.-based Celgene Inc.'s Thalomid (thalidomide, which goes for about $6,000 to $10,000 per patient), approved in 2002, and its analogue Revimid, under development.
Results of the survey suggested 10,000 patients could be treated with Velcade in 2003 and about 13,500 in 2004. Most physicians leaned toward the drug as second-line or salvage therapy. Excluding newly diagnosed patients from projections based on the poll results, about 8,500 patients could be treated in 2003 and 10,500 in 2004, according to MEDACorp.
Giusti, a multiple myeloma patient whose disease has stabilized and whose background includes work in the pharmaceutical industry, noted that patients with the disease have long waited for another treatment option.
"We didn't have anything new for decades," she said, adding that Velcade seems more promising than previously available therapies, and could have value beyond its current label.
"I think it's going to move forward early," she said.