Orphan drug approved for CF.
The Food and Drug Administration has approved Cayston to improve respiratory symptoms in cystic fibrosis patients with Pseudomonas aeruginosa.
The orphan drug was seen as an urgently needed agent to treat the respiratory and pulmonary symptoms of CF, which led the FDA's Anti-Infective Drugs Advisory Committee to overwhelmingly support approval of Cayston (aztre-onam 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 misgivings about missing data and negative regimen effects in the two pivotal trials.
The approval was lauded by the Cystic Fibrosis Foundation as "the first new inhaled antibiotic approved for use in cystic fibrosis in more than a decade."
The foundation also is 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). The call center will offer help to people with cystic fibrosis and members of their care team with insurance verification, referral to participating specialty pharmacies, claims support, and assistance with copayments.
Gilead also is establishing a program designed to minimize barriers to access for Cayston for uninsured, privately insured, and government-insured people.