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Vertex Pharmaceuticals: European Recommendation For Kalydeco Additional Mutations

June 29, 2014

Orphan Druganaut Blog

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…

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