$3B brand challenge – Prolia is one to watch in 2010

The bar is continuing to be raised for new bio-pharma brands by investigators, regulators, insurers, and investors.

One example to keep our eyes on in 2010 is Amgen's bone-loss treatment brand Prolia (denosumab).

In October, the FDA rejected six NDAs, saying it wanted more trials.

But the rewards of getting it right are still significant.  One report says the postmenopausal osteoporosis treatment market, where Prolia is closest to approval, is worth three times more than all the rest combined. And the biggest opportunity is yet to come, if the drug gains approvals for the reduction of fracture risk in various cancers and for the prevention of bone metastases.

Bone loss is a huge and growing medical problem: Up to 50% of women and 30% of men will suffer an osteoporosis-related fracture in their lifetimes, according to the International Osteoporosis Foundation. If the FDA approves Prolia for those uses, the drug could bring in as much as $3 billion a year, analysts predict.

At a time when the country is focused on reforming health care, however, some experts are asking an important question: Can the U.S. afford to protect the bones of millions of aging baby boomers?

In Business Week, Mayo Clinic professor of medicine Dr. Sundeep Khosla questioned whether Amgen's drug is that much better than older, more affordable alternatives, such as Merck's Fosamax, now available as a generic for as little as $100 a year. Amgen has not talked about pricing, but because Prolia is a biologic drug, it will likely cost more than even the priciest alternatives, such as Roche's Boniva and Novartis's Reclast, which cost up to $2,000 a year. "If there's only a marginal difference, you really need a reason not to use those older drugs," says Khosla.

Denosumab performed well in two studies published in the New England Journal of Medicine on Aug. 11. In post-menopausal women with osteoporosis, it reduced hip fractures by 40% and spine fractures by 68%, when tested against a placebo. Yet Amgen has done just a handful of trials comparing denosumab to currently marketed osteoporosis drugs. And those trials only measured bone density--not fracture risk. Without more definitive studies, doctors may choose to stick with old standbys.

Amgen is playing up the fact that Prolia requires nothing more than a twice-yearly injection. Other bone treatments require intravenous infusions, and Fosamax is a pill that's taken weekly or daily. As for the cost, Dr. Roger M. Perlmutter, Amgen's executive vice-president for research and development, said in an interview last June, "We don't intend to be exploitative. We want the value proposition to be clear."

Prolia may be the biggest marketing challenge the 29-year-old biotech has ever faced. The market for osteoporosis drugs is $5 billion but shrinking as insurers push patients to switch to generic Fosamax. That has sparked a marketing war: In 2008, Roche spent $92 million advertising Boniva, according to TNS Media Intelligence, while Novartis spent $53 million pitching Reclast. Amgen will have to put plenty of promotional muscle behind Prolia to convince doctors -- and insurers -- that the drug is a cut above those treatments.

At year’s end, Prolia received recommended for approval by The Committee for Medicinal Products for Human Use of the European Medicines Agency.