On May 8th, 2019 the Pulmonary-Allergy Drugs Advisory Committee (PADAC) convened to review the new drug application (NDA) #202049 for Bronchitol (mannitol inhalation powder) sponsored by Chiesi USA, Inc. Bronchitol, an inhaled dry powder form of mannitol, is intended for the management of cystic fibrosis in adults to improve pulmonary function in conjunction with standard therapies.
Cystic fibrosis is a life-shortening genetic disease. An estimated 30,000 people in the United States are diagnosed with cystic fibrosis. This autosomal recessive disease is a multi-system disorder that affects the airways, exocrine pancreas, GI tract, and reproductive system. There is no cure for cystic fibrosis and current treatments are primarily aimed at management of respiratory complications.
Bronchitol is a sugar alcohol administered by a portable, easy-to use dry powder inhaler. The drug is a naturally occurring osmotic agent that improves lung function by increasing clearance of airway mucus. Three randomized, double blind, controlled phase 3 clinical studies evaluated Bronchitol for improvement in lung function.
Two discussion questions and three voting questions were presented to the committee. The FDA panel voted on whether the applicant provided substantial evidence of Bronchitol’s safety and efficacy in adult cystic fibrosis patients. The panel voted 9 YES to 7 NO. The vote was split for a variety of reasons. The panelists in support thought Bronchitol’s clinical program adequately demonstrated efficacy and addressed safety concerns. The members who voted “NO” were concerned with an increased risk of pulmonary exacerbations and the limited long-term data.
The agency is not obligated to follow its panels’ recommendation, but normally does so. The approval decision will be made in early 2020.
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This post was authored by Sharon Christian. Sharon is a summer intern at ISS and an undergraduate student at Rutgers University.
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