Blockbuster or bust.
Those are the stark outcomes facing Pfizer Inc.'s Exubera, the first inhalable insulin that will arrive on most pharmacy shelves as early as next month.
Pfizer and some industry analysts have said they believe inhaled insulin -- delivered through a small pump about the size of a flashlight and filled with dry powdered insulin packages -- will be quickly adopted by some of the nation's 14.6 million diagnosed diabetes and could account for up to $2 billion a year in sales. That would make it a blockbuster drug at a time when the pharmaceutical industry is having a hard time finding new medications.
The treatment might coax those who resist taking insulin because of their discomfort with needles to get treated. Up to a fifth of diagnosed diabetics do not take insulin or pills as they should, the American Diabetes Assn. estimates. But there's evidence of mounting resistance among doctors and some patients to the new diabetes drug.
The main reason: some doctors worry that the long-term effects of inhaled insulin on the lungs hasn't been studied sufficiently. Research has shown that inhaled insulin causes slight declines in air capacity in the lungs.
Those findings led the Food and Drug Administration to recommend that smokers and asthma sufferers not take the drug. The agency has requested Pfizer to conduct additional research that isn't expected to be completed for several years. It also has not yet approved the medication for children.
"There are still questions about the long-term safety of this product," said Dr. Anastassios Pittas, an endocrinologist and assistant professor of medicine at Tufts University School of Medicine in Boston who was part of Pfizer's initial rollout to a small number of doctors last fall. Pittas said he had prescribed the drug to only two of his patients who requested it and that many of his colleagues also weren't widely prescribing the drug.
Another potential hurdle for wider uptake of the drug: its price. The pump and insulin packets cost about $2,100 a year, a third more than injectable insulin alone and slightly more than the oral medications used by those with a less severe form of the disease.
Many insurance plans are paying for the drug, said an Exubera spokesperson, but some are holding out because they aren't convinced the convenience is worth the higher cost. British and German regulators have recommended against insurance coverage on the grounds that Exubera doesn't treat an unmet medical need.
The FDA approved Exubera a year ago as the first novel way to get insulin in the body ever since the hormone was discovered in 1921. For decades, researchers have been searching for alternative ways for diabetics to get the insulin needed to control their blood sugar levels.
About 5 million Americans inject insulin today, but adherence among patients to medication schedules varies considerably. That's one reason that complications from the disease, such as blindness, kidney failure and amputations, are increasing dramatically.
New York-based Pfizer is expected to announce Monday that it will begin broadly rolling out Exubera across the country over the next few weeks. Because the world's biggest drug maker began only a limited release last fall, most patients haven't had access to it.
Dr. Michael Berelowitz, an endocrinologist and Pfizer's global medical senior vice president, said the drug maker had been researching inhaled insulin for more than a decade and had tested it in more than 5,000 patients. San Carlos-based Nektar Therapeutics developed the inhalers and the powdered insulin formulation for Exubera and receives a royalty on sales.
But Berelowitz acknowledged that sales from the company's early launch late last year had been disappointing and said Pfizer believed adoption would rise quickly as more doctors and patients got comfortable with the new technology.
"This is a major innovation that fills a real medical need," he said. "We're confident the drug is both safe and effective."
There's a lot of money at stake. The number of people with Type 2 diabetes, which is the result of obesity and little to no exercise -- and is sometimes referred to as "diabesity" -- could reach 40 million within a decade. That's why the annual market for diabetes drugs is expected to increase from $15 billion today to $25 billion over the same period.
Three other inhalable insulin products, Denmark-based Novo Nordisk's AERx drug, Indianapolis-based Eli Lilly & Co.'s AIR Insulin System and Valencia-based MannKind Corp.'s Technosphere, are in late-stage development and expected on the market in a few years.
But some diabetics, including Mark Stegemann, a 34-year-old television writer in L.A. , are reticent to try new products that don't show a clear benefit over medication they inject every day that works well for them now.
Stegemann, who was diagnosed three years ago, says he is so used to injecting himself up to twice a day that he no longer notices doing so and doesn't intend to switch to inhaled insulin.
"With all the problems and unknowns with things like [Merck & Co.'s painkiller drug] Vioxx coming out, and the fact that diabetes alone can screw up my body in so many different ways, I don't want to take a risk of injuring my body even more," he said.
Les Funtleyder, a healthcare analyst with Miller Tabak & Co., said Pfizer had better hope it can persuade other patients to see things differently. After a disappointing year in which key products went off patent and promising drugs under development were withdrawn, the company needs a hit, he said.
As early as Monday, the company is expected to lay out a turnaround strategy that is likely to include job cuts and potential plans to acquire smaller biotech companies with promising drug pipelines.