Nektar gets orphan drug designation for inhalation powder for pulmonary fungal infections

By Lisa Kerner

Erie, Pa., Feb. 14 - Nektar Therapeutics said the Food and Drug Administration has granted orphan drug designation to the first amphotericin B inhalation powder for prevention of pulmonary fungal infections in patients at risk for aspergillosis due to immunosuppressive therapy.

Orphan products are developed to treat diseases or conditions that affect fewer than 200,000 people in the United States, according to a company news release. The designation gives Nektar a seven-year period of marketing exclusivity for its product.

Nektar conducted two phase 1 trials, using its proprietary pocket-size inhaler, and said it has long-term toxicity studies underway to support pivotal trials planned for early next year.

Immunosuppressed patients - those receiving either organ or stem-cell transplants or chemotherapy or radiation therapy for hematologic malignancies - commonly develop fungal infections in their lungs, which spread throughout the body (aspergillosis).

Inhaling amphotericin B inhalation powder prior to developing an aspergillosis infection may reduce the incidence of infection and its results, the company said.

Nektar developed the amphotericin B inhalation powder to target the lungs directly with the potent, broad-spectrum, antifungal drug. With this method, the company seeks to eliminate potential systemic toxicities associated with current formulations of amphotericin B delivered intravenously.

"In the United States, more than 75,000 immunocompromised patients annually are at risk of developing often fatal and costly fungal infections in the lungs," Nektar senior vice president, research and development Dr. David Johnston said in the release.

"There are no approved pharmaceutical therapies to prevent fungal infections like aspergillosis, caused when Aspergillus, a widely prevalent genus of molds, infects the lungs and invades the body, causing systemic infections that are very difficult to cure and are associated with an extremely high mortality rate," he continued.

Nektar presented pre-clinical data in December that indicated the inhaled form of amphotericin B provided a statistically significant improvement in the survival of immunosuppressed rabbits challenged with a pulmonary dose of Aspergillus, the release stated.

Separate toxicology studies revealed little or no pulmonary toxicity when animals were administered 10 times or more than the expected amphotericin B powder dose for humans, with no systemic toxicity and low amounts of the drug in the bloodstream.

Pivotal trials are on target to begin in early 2007.

Nektar develops drug-delivery technologies and differentiated therapeutics for biotechnology and pharmaceutical companies. The company is based in San Carlos, Calif.

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