The FDA approved Vertex's second drug today when it ratified Kalydeco (ivacaftor) for sale to cystic fibrosis patients with the G551D mutation. These patients represent about 4% of the US CF population or about 1200 patients. There are another 1000 such patients in Europe. Vertex is seeking approval in the EU by Qtr 3 in 2012.
Kalydeco represents the first drug that actually corrects for the dysfunctional CF protein CFTR that results in disease for these patients. Genetic tests will reveal which patients have the G551D mutation and therefore will be good candidates for the drug. In clincal trials, Kalydeco resulted in significant improvement to lung function, reduced side effects and improved quality of life. The drug will cost $294,000 per year per patient. Analysts predict peak sales of Kalydeco of around $550 M annually. The drug also carries orphan status.
There is real excitement that this targeted therapy made it thorugh the FDA in 3 months -- a record. This is also the 2nd personalized medicine drug in approved by the FDA over the past few days. It shows the high-level agency interest in programs of this sort and their favorable disposition.
"Kalydeco is an excellent example of the promise of personalized medicine --- targeted drugs that treat patients with a specific genetic makeup," said FDA Commissioner Margaret Hamburg.
The drug was part of a 13 year development program with the CF Foundation, which spent almost $70 M towards development. The CFF will now also benefit with this approval by collecting royalties that start in "high single digits" and could reach as high as 12 percent. A first and a win for this type of collaboration. See Xconomy, Fierce Biotech, and PharmTech Talk.
Posted by Bruce Lehr Jan 31st 2012.