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Vertex Pharmaceuticals: Another Step Forward For Kalydeco

February 22, 2014


On February 21st, Vertex Pharmaceuticals announces that the FDA approves a supplemental New Drug Application (sNDA) for orphan drug Kalydeco (Ivacaftor) for people with Cystic Fibrosis (CF), ages 6 and older, who have one of the 8 additional mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene :

•   G178R
•   S549N
•   S549R
•   G551S
•   G1244E
•   S1251N
•   S1255P
•   G1349D.

Kalydeco receives approval from the FDA in January 2012 for CF patients, ages 6 and older who have at least one copy of the G551D mutation. Thus, Kalydeco is currently approved for 9 mutations. The new approval affects approximately 150 in the United States.

The sNDA approval is based on previously announced data from a Phase III, 2-part, randomized, double-blind, placebo-controlled, cross-over study of 39 CF patients who have one of the above listed 8 mutations + the G970R mutation. Based on this study, efficacy of Kalydeco for patients with the G970R mutation could not be shown to support approval for this particular mutation. Approximately 10 patients worldwide have the G970R mutation, with two in the United States.

Regulatory submissions are made in Europe, Canada, and Australia for approval of Kalydeco for treatment of CF patients, ages 6 and older with these additional mutations. Approximately 250 have these additional CF mutations in Europe and Australia.

Kalydeco Regulatory Approval History

•   Approval for people with CF, ages 6 and older, who have at least 1 copy of the G551D mutation in the CFTR gene :

1.   FDA in January 2012

2.   EMA in July 2012

3.   Health Canada in November 2012

4.   TGA (Australia) in July 2013

•   FDA approval in February 2013, for people with CF, ages 6 and older, who have mutations in the CFTR gene: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.

In other Kalydeco news, according to an online February 21st article from Adam Feuerstein, Vertex Pharmaceuticals has switched around the primary and secondary endpoints of two important Phase III clinical trials (Kalydeco + Lumacaftor (VX-809) for patients, ages 12 and older, who are homozygous for the F508del mutation) :


Results from these 2 studies are expected in mid-2014. Also, according to the article, “Results from these 2 studies ….. have the potential to deliver $6 billion or so in new sales to the company.”

Please Note: FDA Official Logo from FDA website.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.


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