An FDA advisory committee recommended approval of Nektar Therapeutics Inc.'s inhaled insulin product, Exubera - offering hope to many Type I and Type II diabetics who, for more than 80 years, had to stick themselves with needles to control their glucose levels.

Exubera is a product that Wall Street thought would reach the market earlier this decade, but concerns over its effects on lung function delayed its introduction and required another lengthy trial.

"They needed to better characterize those changes," said Robert Hazlett, managing director of Atlanta-based Suntrust Robinson Humphrey, from his offices in New York. "It took another long-term safety study with rigorous data collection methodologies to demonstrate convincingly that there was not a dramatic effect on lung function when you use the product."

The FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted 7-2 Thursday to approve Exubera. Its PDUFA date is expected for late October, and if approved, a launch could occur in early 2006.

Although it unanimously voted in favor of the safety data presented, the committee raised some concerns about the product's use in patients with underlying lung disease and the ability of patients to titrate the dose correctly.

If approved, Exubera would be indicated only for adults who are non-smokers and those without lung disease, such as asthma or chronic obstructive pulmonary disease.

For San Carlos, Calif.-based Nektar, Exubera holds great potential. Sales could easily exceed $2 billion five years after launch, Hazlett said. Eventually, the product could cannibalize day-to-day injectable insulin.

"We have some products on the market, but we get small royalties on those," said Chris Searcy, Nektar's vice president of corporate development and the head of investor relations. "This is our flagship. This is our most important product. Not only is it important to us, but we feel deeply and passionately that this has potential to help lots and lots of patients."

One patient advocate who attended the committee meeting Thursday said that marketed Exubera would be a "siren call that is irresistible" to diabetics.

Searcy said the target market includes about 5 million people in the U.S. who either have trouble taking enough insulin because of the number of injections required, or those who are unwilling to go on insulin because of the injections and whose diabetes is poorly controlled with oral medications.

About 90 percent to 95 percent of the 18 million diabetics in the U.S. have Type II diabetes, and 67 percent of them have blood sugar levels that are not controlled or are above the recommended national treatment guidelines.

If Exubera is approved in Europe, the target market would be 10 million people, Searcy said. European regulatory authorities accepted for review the Exubera application in 2003, but have not ruled on it.

Founded in 1990, Nektar (then called Inhale Therapeutic Systems Inc.) began working on Exubera shortly after inception, doing its first clinical trial in 1994. In early 1995, it partnered the product with Pfizer Inc., of New York, which signed a co-development and co-promotion deal with Paris-based Sanofi-Aventis Group in 1998. Pfizer and Sanofi-Aventis share worldwide rights to the product, and Nektar will receive an undisclosed royalty on sales, as well as manufacturing rights on a cost-plus basis.

Nektar is responsible for all of the devices and for half of the powder processing, while Pfizer is responsible for half of the powder processing and all of the powder filling.

In diabetics, Exubera mimics the normal physiological insulin response to meals by being absorbed into the bloodstream to reduce spikes in glucose levels. The product is inhaled into the lungs prior to eating. If approved, it would be the first non-injectable insulin available in the U.S. since the discovery of insulin in the 1920s.

"Rapid acting [injectable] insulins will certainly be under pressure from this product," Hazlett told BioWorld Today. "That being said, not all patients will be able to use this product, at least initially, but we think the compelling nature of the ability to lessen the burden of injections will be very appealing to patients."

Hazlett added that he expects Pfizer will try to expand the label into younger patients and into those with serious lung conditions.

Nektar's current products on the market, all partnered, are based on the company's PEGylation technology. They include Neulasta (Amgen Inc.) for infection caused by chemotherapy, Somavert (Pfizer Inc.) for acromegaly and Pegasys (F. Hoffmann-La Roche Ltd.) for hepatitis C, to name a few. It also has several products in development with partners.

Nektar's stock (NASDAQ:NKTR) closed $19.59 Friday, up 45 cents after trading as high as $21.52. In addition, an institutional investor elected to exercise its option to acquire 454,803 shares for $8 million, which is added to the $24 million in stock the investor already had acquired in August.

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