Nektar Therapeutics (NKTR)

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Diabetics get some good news: FDA approves inhaled insulin
Pfizer and San Carlos company collaborate to produce powdered form of hormone
Justin Gillis, Washington Post
Saturday, January 28, 2006

Washington -- The Food and Drug Administration on Friday approved an inhaled form of insulin, the first new way to get that hormone into the body since it was discovered in 1921 -- and a new treatment option for many of the 21 million Americans with diabetes.

The approval fulfills an arduous scientific quest that spanned most of the 20th century and spilled over to the 21st. And it marks the biggest change in diabetes treatment in decades, one that doctors hope will lure a fair slice of the American population into their offices to talk about controlling blood sugar. The product poses long-term safety questions, though, and it's not clear whether it will be more expensive than standard insulin.

Millions of Americans need treatment with insulin but don't get it because it involves frequent, painful needle sticks and injections. About 5 million take the hormone, but a high proportion inject themselves too few times during the day because it's so inconvenient. Doctors hope inhaled insulin will overcome some of that resistance, helping diabetics ward off a slew of medical problems that afflict those who don't control their disease.

Studies show the new product, to be sold by Pfizer Inc. under the brand name Exubera, works and appears to be safe with short-term use. Patients who have used inhalers told researchers they prefer them to needles by a wide margin, according to studies sponsored by Pfizer.

"I'm just flabbergasted at the number of people who really do seem to want this, and want it substantially," said Jay Skyler, a University of Miami doctor and one of the nation's leading diabetes experts.

However, inhaled insulin causes minor declines in how much air the lungs can hold. Scientists consider that a signal that long-term use could pose risks, although that could take years to sort out. The FDA recommended that smokers and people with some types of lung disease, including asthma, avoid using the product. Exubera is approved only for people 18 or older, though studies in children are under way.

Pfizer said the product wouldn't be available in most pharmacies until June or July. Exact prices haven't been set, but Vanessa Aristide, a Pfizer spokeswoman, said the product would be "priced competitively" with injected insulin. Pfizer is first to market with such a product, but others are under development.

Pfizer's partner in the development of Exubera is a Bay Area biotechnology firm, Nektar Therapeutics of San Carlos. The technology to produce the inhalant, powdered form of insulin was the fruit of 20 years of research by scientists led by Nektar's co-founder, John Patton.

The team had to produce insulin particles small enough to be propelled by a hand-held inhaler and to penetrate deep into the lungs, where the drug could be absorbed into the bloodstream. The powdered drug also had to be stable at room temperature.

Nektar said it drew inspiration from engineering tactics used in food processing, the music industry, diesel carburetor design and scuba equipment manufacturing.

"Exubera would not have been possible without Nektar's innovative scientists and engineers and also our partner, Pfizer, who worked with us and remained committed to our original dream of delivering this medical breakthrough to patients," said Patton.

Friday's decision confronts millions of Americans -- diabetics make up 7 percent of the population -- with a complicated new strategic problem, requiring them to figure out how much long-range risk they're willing to incur for the convenience, and possibly greater disease control, of using inhaled insulin.

"The issue comes down to: How do we all deal with uncertainty?" said Robert Rizza, a diabetes specialist at the Mayo Clinic and president of the American Diabetes Association. "We just don't know what the long-run safety record will be. Each person will now need to think very carefully about the potential benefits and the risks for them."

The human body burns a simple sugar, glucose, in much the way a car burns gasoline. But the level of this essential fuel in the blood must be tightly controlled, because too much can wreck tiny blood vessels and cause other problems. The pancreas monitors glucose levels and releases insulin, a hormone that signals cells to absorb the sugar.

Diabetes is a pervasive group of diseases in which this fundamental life process has gone awry. Some people's bodies don't make insulin at all, and they must take it as a medicine or die. But the vast majority of diabetics have a milder form of the disease in which their bodies make too little insulin, resist its effects or both.

Diabetes can be controlled in both groups, but it isn't easy. Diet and exercise are important. Pills help some people, but many others need supplemental insulin, which cannot be given as a pill. They have to prick their fingers to test blood-sugar levels and inject themselves repeatedly throughout the day with insulin, or wear pager-sized insulin pumps that deliver the hormone through tiny needles.

The sheer tedium of the task gets diabetics down, and overall, they do poorly at it. A third of Americans with diabetes don't even know they have the disease, the government estimates, and many others fail to achieve adequate control of their blood sugar. The long-term result is a litany of severe medical problems: blindness, impotence, limb amputation, kidney failure, heart attack. The government pegs costs at more than $100 billion a year.

http://www.sfgate.com/cgi-bin/article.cgi?file=/c/a/2006/01/28/MNGAIGURAH1.DTL