The Cambridge, MA-based biotech released new data from a late-stage clinical trial today showing that its CF drug, ivacaftor (Kalydeco), improved the lung function of patients aged six and older who have at least one so-called non-G551D gating mutation.
Vertex reported ivacaftor improved these patients’ lung function by 10.7 percentage points on average when compared with a placebo. The results from the 39-patient study were statistically significant, meaning they weren’t likely the result of chance. Based on the numbers, Vertex plans to file applications to regulators in the U.S. and Europe in the second half of the year to approve ivacaftor as a single drug given on its own for people with cystic fibrosis who have at least one non-G551D gating mutation.
Only about 400 patients worldwide have this specific genetic mutation, but even so, it represents another building block for Vertex to scale up ivacaftor. The drug is already approved in the U.S. and European Union for use in cystic fibrosis patients that have the G551D gene mutation. About 2,000 patients worldwide, or about 3 percent of the total population of 70,000 cystic fibrosis patients, have that abnormality. Thus, should Vertex win regulatory approval for the new group, it could boost its potential worldwide market by about 20 percent.
While 400 patients might sound like a small group of people, each step ahead in expanding the reach of ivacaftor is important for Vertex’s business. The drug costs more than $300,000 per year, per patient, and is taken chronically by people with increasing life expectancies. Vertex reported separately today that ivacaftor had $160.8 million in sales from the drug during the first half of 2013, and $99.0 million for the second quarter—beating consensus estimates for the quarter of $74 million, according to ISI Group analyst Mark Schoenebaum.
Those numbers caused Vertex to raise its 2013 sales estimates for the drug to between $345 million and $360 million. Vertex previously forecasted sales this year of between $300 million and $340 million.
Vertex’s shares were up a little over 2 percent, to $81.50 apiece, in after hours trading.