Millennium's Short Road For Velcade Approval On 13 May 2003 The
Food and Drug Administration yesterday approved Millennium Pharmaceuticals'
cancer drug Velcade to treat multiple myeloma, a deadly blood cancer.
The FDA approved Velcade in a record four months. Roughly 14,000 people
get the disease annually, and most die within five years. Impressively,
Velcade was invented less than eight years ago. Usually, getting a drug
from the laboratory to the market takes a decade and a half.
The FDA approval comes without a controlled clinical trial which
is usually required for a regulatory OK. But in studies, Velcade clearly
helped a third of multiple myeloma patients who had failed two or more
previous therapies--a stunning result in such a lethal disease. "It's
the first drug of its class," says Ken Anderson, director of the Jerome
Lipper Multiple Myeloma Center at the Dana-Farber Cancer Institute,
"and we are hopeful that more potent, second-line derivatives are coming
in the pipeline. We're anxious now to test Velcade together with conventional
as well as other novel therapies, as well as use it to treat patients
with myeloma earlier in their disease course." Velcade was concocted
at a tiny, Cambridge, Mass.-based startup called ProScript in 1995 by
a team led by chemist Julian Adams, who had cooked up the AIDS drug
Viramune at Boehringer Ingleheim. Velcade targeted an obscure complex
of enzymes, called the proteasome, which acts as a kind of cellular
maid. If the proteasome stopped its house-cleaning duties, scientists
reasoned, cancer cells would give up.
By 1998, the drug, then known as PS-341, had advanced into early-stage
human testing. But ProScript ran low on money. In 1999, the firm
was folded into another Cambridge company, LeukoSite, which was purchased
four months later by Millennium (nasdaq: MLNM - news - people ). Most
drugs would have been lost in the shuffle, but Velcade had two advantages:
strong data in human beings, and the enthusiastic Adams, who has since
become Millennium's director of target discovery.
Already, a coalition was forged between Adams and academics at the
Dana-Farber Cancer Institute and the National Cancer Institute. Millennium
Chief Executive Mark Levin made Velcade his company's most-funded drug.
.
"This is an exceptional model in public-private partnership," says
Kathy Giusti, head of the Multiple Myeloma Research Foundation. The
MMRF poured $2 million into Velcade's development. Giusti, who was diagnosed
with multiple myeloma in 1996, is a former sales executive at Merck
(nyse: MRK - news - people ) and G.D. Searle, which is now part of Pfizer
(nyse: PFE - news - people ). She is aware that drug companies often
balk at relatively small diseases.
But she points out that her organization and others have been eager
to help biotech companies like Millennium and Celgene (nasdaq: CELG
- news - people ) study myeloma, and that studying multiple myeloma,
which can be measured with a simple blood test, can be used as a launching
pad for understanding how drugs work and how they might be used in other
cancers. "That's exactly what Millennium did," Giusti says.
"They were very smart." Now, Millennium is raring to go. Barry Greene,
Millennium's general manager for oncology, says that the company
has trained 75 salespeople to sell the drug, despite the early timing
of the approval.
"We were confident when we filed the new drug application," he says.
The main task needed before getting the drug out is printing up the
drug's FDA-approved label and shipping it with the vials, Greene says.
Further studies of Velcade are still needed. A controlled trial verifying
the current results should be completed in 2005. In a few years, Velcade
could be tested as a frontline treatment in previously undiagnosed myeloma
patients. Velcade will also be tested in other cancers, and genomic
tests included in late-stage trials could make it possible to pick out
those who will respond to the drug from those who will not. "I think
it's years off," says Dana-Farber's Anderson. "We believe the broad
opportunity really is a tremendous opportunity for the financials of
Millennium," Greene says.
Millennium has previously forecast that it would break even in 2006.
That forecast factored in an approval for Velcade. Roughly a quarter
of the 45,000 Americans with multiple myeloma are eligible for treatment
with Velcade under the drug's current label. On average, treating one
patient with the drug costs $20,000.