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Vertex Pharmaceuticals Announces Kalydeco’s Ambitious New Program

February 27, 2013

On February 26, 2013, Vertex Pharmaceuticals announces the initiation of a global pivotal Phase III development program in Cystic Fibrosis (CF). The new program is for fixed-dose combinations of VX-809 (Lumacaftor) and orphan drug Kalydeco (Ivacaftor) for CF patients who have two copies of the F508del mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. This is the most common form of CF.

Vertex Pharmaceuticals plans on two 24-week Phase III clinical trials, TRAFFIC and TRANSPORT, to evaluate two different doses of VX-809 in combination with Kalydeco, in CF patients ages 12 and older. Both clinical trials will enroll 500 patients at 200 sites. Also, both trials will have two treatment groups that evaluates VX-809 in combination with Kalydeco compared to a placebo group. Results from both clinical trials may be ready by the end of 2013 or during the first half of 2013. If the results are positive, then Vertex Pharmaceuticals plans on filing for US regulatory approval in 2014.

The additional good news is that Vertex Pharmaceuticals expects 24-week safety and efficacy data from both studies, plans to submit both a FDA New Drug Application (NDA) and Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), pending the clinical trial results.

Also, Vertex Pharmaceuticals plans to do a pharmacokinetics and safety study to evaluate VX-809 in combination with Kalydeco in CF patients, ages 6 – 11, who have two copies of the F508del mutation. The data from this study will be used for registration in patients ages 6 – 11 in the US. The company is in discussions with EU regulatory agencies for patients in this same age group.

The initial approval of Kalydeco is for CF patients, ages  and older with the G551D gene only. Approximately 4 % of the global CF population have the G551D gene. By seeking regulatory approval for CF patients who have two copies of the F508del mutation, this will increase the CF patients who qualify for treatment with Kalydeco to nearly 50% of about 70,000 global patients.

Kalydeco sales for 2012 are $172 million. The CF program is important for Vertex Pharmaceuticals future as sales of the company’s Hepatitis C drug Incivek has decreased.

In January 2013, Kalydeco receives the first two FDA Breakthrough Therapy Designations for :

1) Monotherapy for potential additional indications beyond the current approval for CF patients 6 and older with the G551D mutation

2) Combination therapy with VX-809.

References

Vertex Pharmaceuticals February 26, 2013 Press Release

Vertex Pharmaceuticals February 26, 2013 Webcast

Vertex Pharmaceuticals Pipeline

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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