If approved, up to 10,000 people in Europe, would be newly eligible for the medicine
Vertex Pharmaceuticals has announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor), in people aged 12 and older with the most common genotypes of cystic fibrosis (CF).
The positive opinion adopted for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with Kalydeco (ivacaftor) is to treat people with CF, aged 12 and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
If approved, up to 10,000 people in Europe with CF, ages 12 and older who have one F508del mutation and one minimal function mutation, would be newly eligible for a medicine that targets the disease-causing protein defect.
Additionally, people 12 years of age and older who have two F508del mutations and who are currently eligible for one of Vertex’s other EMA-approved cystic fibrosis medicines would be eligible for the new triple combination regime.
Since 2017, eligible patients with CF in Ireland have had access to Vertex medicines via the long-term reimbursement agreement that was developed with the Health Service Executive (HSE) and Irish Government.
As a result, if approved, people living with CF in Ireland ages 12 and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the CFTR gene, would be among the first in Europe to receive reimbursed access to the medicine.
“If approved, this would be the first CFTR modulator for people with one F508del mutation and one minimal function mutation and would bring additional benefit to people with two F508del mutations,†said Carmen Bozic, MD, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.
“This milestone brings us one step closer to delivering this innovative CF medicine to those who are waiting, and toward our ultimate goal of providing a therapeutic option for every person with this rare and devastating disease.â€