Bronchitol AdComm Shows Difficulty Of Following In Kalydeco’s Shadow
Comparisons between the robustness of the efficacy data for dry powder mannitol and Vertex’s ivacaftor were difficult for FDA to avoid during the Pulmonary-Allergy Drugs Advisory Committee’s Jan. 30 review of Pharmaxis’ cystic fibrosis drug.
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Bronchitol is set to make its second appearance before a US FDA advisory committee, and Chiesi hopes to rely on the drug’s novel mechanism of action and the unmet need in cystic fibrosis to overcome the agency’s concerns about the underlying clinical data.
Cystic Fibrosis Market Snapshot: Disease-Modifying Drugs Elusive 24 Years After Discovery Of Root Cause
The discovery in 1989 of the underlying genetics causing cystic fibrosis was expected to spur broad development of disease-modifying therapies. As of 2013, however, only one such drug is on the market, for a small subset of CF patients, and a few other candidates are in clinical development.
Label expansion efforts for Vertex’s cystic fibrosis therapy will be claims taken through FDA’s new review pathway; the initial product could reach more patients more quickly and serve as a guide to how the agency will handle drugs earmarked for streamlined development and approval.