Vertex Presenting 15 Cystic Fibrosis Abstract at Upcoming Conference

James Radke

Vertex Pharmaceuticals announced they will present a staggering 15 abstracts at the 28th Annual North American Cystic Fibrosis Conference (NACFC) in Atlanta, GA, October 9 - 11, 2014.  

Kalydeco plus lumacoftor

Key among the presentations will be ones focused on data from the Phase 3 TRAFFIC and TRANSPORT studies of the company’s pipeline drug lumacaftor in combination with their approved drug Kalydeco (ivacaftor) in people with cystic fibrosis who have two copies of the F508del mutation.  

Right now, Kalydeco can treat about 2000 patients with cystic fibrosis and has the potential to reach about 7,000.  Vertex’s Lumacaftor or VX-809 in combination with Kalydeco is being tested on patients with  F508del homozygous who account for over 28,000 patients with cystic fibrosis.

These presentations include:
Other presentation association with the combination Kalydeco and lumacaftor include:

Kaledcyo monotherapy

Data from studies involving Kalydeco in different genetic mutations of cystic fibrosis are also being presented. At present, Kalydeco (150 mg tablets) is indicated for the treatment of cystic fibrosis in patients age 6 years and older who have  one of the following mutations in the  CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N,  or S549R.

Multiple Phase 2 and Phase 3 studies of Kalydeco monotherapy will be presented,  including the first presentation of the Phase 3 results of Kalydeco treatment in children ages 2 to 5. Presentations related to Kalydeco monotherapy include:

Kalydeco plus VX-661

VX-661 is another corrector being tested in combination with Kalydeco that is being explored in both F508del homozygous and heterozygous patients (>45,000 patients).

About Cystic Fibrosis

In the United States, there are approximately 30,000 people living with cystic fibrosis (70,000 worldwide).  Cystic fibrosis is caused by a mutation in the gene for the protein cystic fibrosis transmembrane conductance regulator (CFTR). The most common mutation is  F508del; however, there are over 1500 other mutations that can produce cystic fibrosis.

Disruption of the protein CFTR leads to secretion/mucus build up in organs, especially in the lung.  Kalydeco is the only treatment currently available that addresses the underlying cause of cystic fibrosis and it is approved for persons with the G551D mutation as well as several other mutations that account for about 2,000 of the 70,000 patients with cystic fibrosis.

Patients with cystic fibrosis must regularly clear their airways to avoid mucus buildup (with physical therapy techniques and/or inhaled medications). The chronic condition also benefits from regular exercise to clear mucus in the lungs and dietary enzyme supplements to improve absorption.  For more information about cystic fibrosis, visit
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