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Vertex Pharmaceuticals: European Recommendation For Kalydeco Additional Mutations

June 28, 2014

On June 27th, Vertex Pharmaceuticals gets European CHMP’s (Committee for Medicinal Products for Human Use) positive opinion recommending the approval of orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis (CF) patients, ages 6 and older, who have 1 of the following 8 non-G551D gating mutations, in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene:

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

These 8 additional mutations affects approximately 250 patients in Europe. The next step, is for the European Commission (EC), that has the authority to approve drugs for the European Union, to review the CHMP’s positive opinion. The EC usually follows the recommendation of the CHMP and issues a marketing approval within 3 – 4 months. Kalydeco receives approval in Europe in July 2012 for patients with CF ages 6 and older, who have at least 1 copy of the G551D mutation, which is the most common gating mutation.

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. Then in February 2014, the FDA approves a supplemental New Drug Application (sNDA) for Kalydeco, for people with CF ages 6 and older, who have 8 additional non-G551D gating mutations (mentioned above) in the CFTR gene. The approval of the 8 additional mutations, affects approximately 150 in the US. Thus, Kalydeco is currently approved for 9 mutations in the US.

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

5.    Medsafe (New Zealand) in December 2013

6.    Swissmedic (Switzerland) in January 2014.

•   FDA approval in February 2014, for people with CF, ages 6 and older, who have the following 8 mutations in the CFTR gene: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D

•   Health Canada’s approval in June 2014, for people with CF, ages 6 and older, who have the following 9 mutations in the CFTR gene: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D, and G970R

•   European CHMP’s positive opinion in June 2014, for people with CF, ages 6 and older, who have the following 8 mutations in the CFTR gene: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.

Please Note: “Chemlab2” by Tahoenathan (Own work) [CC-BY-SA-3.0] | via Wikimedia Commons.

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

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