A Treatment Suitable for Half of Cystic Fibrosis Patients?

[enniob]

Very promising news out today on at "cystic fibrosis news today" summing up the importance of the recent Vertex combination therapy trials with lumacaftor and ivacaftor (Kalydeco) in patients with cystic fibrosis of the F508del-CFTR mutation.

As they noted in a recent post, lumacaftor is a corrector for the CFTR protein, and ivacaftor is a potentiator for the CFTR protein. Ivacaftor can treat approximately 2,000 cystic fibrosis patients, which account for 4% of the disease population, by activating CFTR on the cell membrane.

By using lumacaftor to help CFTR properly fold, Vertex may be able to help the majority of cystic fibrosis patients, raising the number to 22,000 patients. Shooting even higher is a three-drug cocktail from Vertex that may be able to treat another 17,000.

Jeffrey Leiden, chief executive of Vertex, was quoted in saying:

“It’s going to potentially allow us to get a medicine to 22,000 patients around the world with the most common form of cystic fibrosis,”

To open this treatment for patients with the F508del-CFTR mutation, Vertex may file the combination treatment for approval in the United States and Europe by the end of the year. Ivacaftor is already marketed by Vertex under the name Kalydeco, and lumacaftor is an experimental compound. Jeffery Leiden said:

“It will be the first medicine that treats the underlying cause of the disease for those patients. Every improvement in lung function, every day they stay out of the hospital, every pound they gain, is meaningful.”

A recent blog at CysticFibrosis.com announced that the EU has already given approval for single drug Kalydeco therapy to be tested in Europe on 250 CF patients that qualify, which will include 8 additional mutations including Patients with the G178R, S549N, S549R, G551S, G1244E, S1251N and S1255P. It should not be long before this combination therapy with lumacaftor and ivacaftor (Kalydeco) should also be available in the EU....watch this space.