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ivacaftor (Kalydeco)

Advice

following a full submission assessed under the ultra orphan medicine process

ivacaftor (Kalydeco®) is not recommended for use within NHS Scotland.

Indication under review: treatment of children with cystic fibrosis (CF) aged 2 years and older and weighing less than 25kg who have one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

In an open-label single-arm study, acceptable safety was demonstrated in children aged 2 to 5 years.

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust clinical and economic analysis to gain acceptance by SMC.

This advice takes account of the views from a Patient and Clinician and Engagement (PACE) meeting.

Drug Details

Drug Name: ivacaftor (Kalydeco)
SMC Drug ID: 1134/16
Manufacturer: Vertex Pharmaceuticals Ltd
Indication: Treatment of children with cystic fibrosis (CF) aged 2 years and older and weighing less than 25kg who have one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
BNF Category:
Sub Category: 3.7 Mucolytics
Submission Type: Full submission
Status: Not Recommended
Date Advice Published: 9 May 2016

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