Vertex’s Kalydeco (ivacaftor) Receives EC’s Approval for Infants with Cystic Fibrosis with CFTR Gene Mutation Aged 6 to
Vertex’s Kalydeco (ivacaftor) Receives EC’s Approval for Infants with Cystic Fibrosis with CFTR Gene Mutation Aged 6 to <12mos.
Shots:
- The EC’s approval is based on P-III ARRIVAL open-label safety study assessing Kalydeco in children with CF aged < 24mos. with CFTR gating mutation, demonstrated similar safety profile as observed in other P-III studies and showed improvement in sweat chloride as 2EPs
- Following the approval, children with CF in EU as young as 6 mos. with at least one of the following nine mutations in their CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R will be able to access the therapy treating the underlying cause of the disease
- Kalydeco is an oral CFTR potentiator that enables the longer opening of CFTR protein at the cell surface to improve the transport of salt and water across the cell membrane and is an already approved therapy for CF patients with CFTR mutation aged ≥ 12mos.
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