10 June 2020 - EU regulators have expanded the treatment scope of Vertex' Kalydeco (ivacaftor) to include a new subset of young patients with cystic fibrosis.

Doctors can now also prescribe the treatment for children and adolescents with cystic fibrosis, aged six months and older and weighing at least 5 kg, who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator gene – the most common residual function mutation underlying the condition.

Kalydeco (ivacaftor) will be immediately available to additional eligible patients in Germany and shortly in countries where respective long-term reimbursement agreements have been previously secured, Vertex said, adding that it will also 'work closely' with all other relevant government authorities 'to secure access for eligible patients as quickly as possible'.

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