Novartis CF therapy sails through FDA advisory panel review

Novartis found it relatively easy to convince a lopsided majority of experts on an FDA advisory panel to sign off on its new powdered formulation for an inhaled cystic fibrosis therapy. The inhaled antibiotic is intended to treat lung infections that afflict CF patients, and by a vote of 13 to 1 the outside experts recommended it to regulators for approval.

Novartis ($NVS) has been pushing for an easier form of delivery, replacing the nebulizer that had been required to deliver tobramycin in favor of an inhaler. Overall sales of the drug came close to $300 million last year, Reuters reports, making this a moderate but steady earner for Novartis.

An FDA staff review had raised questions about a high dropout rate among patients recruited for Novartis's study. "A major limitation of Study C2301 is that the primary analysis excluded 41% of the randomized population. The analysis based on 61 (59%) of the original ITT population of 102 patients is seriously limited as the patients who dropped out could be informative," noted the agency in its review. "This limitation could introduce potential biases. The FDA reviewer's sensitivity analysis based on all randomized patients receiving treatment showed a smaller magnitude of treatment effect." 

But the experts seemed content to overlook the data limitations in favor of the improved delivery method. "I think it's wonderful there's an additional drug available. Its importance, as with any new drug, will be determined over time," Johns Hopkins' Dr. Paul Auwaerter noted, according to the Reuters report. 

The FDA, of course, doesn't have to obey the wishes of the panel, but it usually does.

- here's the story from Reuters