The Food and Drug Administration has approved Cayston to improve respiratory symptoms in cystic fibrosis patients with Pseudomonas aeruginosa, a therapeutic area with few meaningful therapies.
The approval comes as no surprise, as the orphan drug was seen as an urgently needed therapeutic to treat the respiratory and pulmonary symptoms of CF, and led the FDA's Anti-Infective Drugs Advisory Committee to overwhelmingly support approval of Cayston (aztreonam for inhalation solution). The drug is manufactured by Gilead Sciences.
Members of the committee said the bar for approval should be set “quite low” due to the lack of meaningful alternatives, despite both the FDA's and the panelists' misgivings about missing data and negative regimen effects in the two pivotal trials.
The approval was lauded by the Cystic Fibrosis Foundation, whose president and CEO Robert Beall said in Gilead's press release, “As the first new inhaled antibiotic approved for use in cystic fibrosis in more than a decade, Cayston therefore represents an important therapeutic option in the care of patients with cystic fibrosis.”
The foundation is also working with Gilead's marketing team to establish the Cayston Access Program, a call center developed with the Cystic Fibrosis Foundation Pharmacy (a wholly owned subsidiary of the Cystic Fibrosis Foundation), which will assist people with cystic fibrosis and members of their care team with insurance verification, referral to participating specialty pharmacies, claims support, and copay assistance.
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