Gilead Prices Cystic Fibrosis Drug Cayston At Slight Premium To Competitor

Gilead Sciences will price its cystic fibrosis drug Cayston at an 8 percent premium to its competitor, Novartis' TOBI (tobramycin).

FDA approved Gilead Sciences' Cayston (aztreonam for inhalation solution) to improve respiratory symptoms in cystic fibrosis patients with Pseudomonas aeruginosa, a therapeutic area with few meaningful therapies.

The $4,427/month price tag will be pushed by a "very small and targeted" sales force, according to a research note from Baird analyst Thomas Russo. Baird predicts Cayston's revenues will reach $224 million in 2012.

It won't be a major product for Gilead, a company with a stronghold of HIV therapies and promising pipeline projects in the related hepatitis C space, but Cayston will be a major advance for the CF community.

The orphan drug was seen as an urgently needed therapeutic to treat the respiratory and pulmonary symptoms of CF, a factor that led FDA's Anti-Infective Drugs Advisory Committee to overwhelmingly support its approval.

Members of the committee said the bar for approval should be set "quite low" due to the lack of meaningful alternatives, despite both FDA and panelist misgivings about missing data and negative regimen effects in the two pivotal trials (¹ (Also see "Gilead's Cayston Picks Up Panel Endorsement, But Not Based On Data" - Pink Sheet, 14 Dec, 2009.)).

The approval was lauded by the Cystic Fibrosis Foundation; CFF 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 working with Gilead's marketing team to establish the Cayston Access Program, a call center developed with Cystic Fibrosis Foundation Pharmacy - a wholly owned subsidiary of the Cystic Fibrosis Foundation, which has been at the head of the trend of disease foundations moving beyond advocacy into more active roles in drug development (² (Also see "Advocacy Foundations Bridging Gap Between Research & Drug Development" - Pink Sheet, 29 Apr, 2005.)).

With the collaboration on the Cayston Access Program, the CFF is now also getting involved in commercialization.

The Cayston call center will assist people with cystic fibrosis and members of their care team with insurance verification, referral to participating specialty pharmacies, claims support and co-pay assistance.

Gilead is also establishing a program designed to minimize barriers to access for Cayston for uninsured, privately insured and government-insured people.

Such steps - coordination with disease advocacy groups, patient assistance programs and outreach efforts - are now a standard part of the repertoire for launching a therapeutic for an orphan disease.

CF is a chronic genetic condition that affects the respiratory and digestive systems of approximately 70,000 people worldwide, including 30,000 people in the U.S. Chronic respiratory tract infection with P. aeruginosa contributes to the decline in pulmonary function, which is often associated with morbidity and mortality among CF patients.

FDA's approval of Cayston comes with a few post-marketing commitments, including a prospective study to assess pseudomonas susceptibility to Cayston over five years (final report is due January 2018); a prospective head-to-head trial versus TOBI (already in progress with completion date May 2010 and submission date September 2010); and a dosing study evaluating three times per day versus two times per day (final report due January 2014).

The drug will be available in the United States by the end of the first week of March, through certain specialty pharmacies.

Cayston, administered at a dose of 75 mg three times daily over a 28-day period, is delivered via the Altera Nebulizer System, a portable, drug-specific delivery device using developed by PARI Pharma.

Cayston was approved in the EU and Canada in September 2009 and was approved in Australia in January 2010. Applications for marketing are currently pending in Switzerland and Turkey.

- Lauren Smith ( ³ [email protected] )