Vertex: CHMP favorable opinion for SYMKEVI® MyPharma Editions

Vertex, the US biotech company, has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA ) adopted a favorable opinion for SYMKEVI® (tezacaftor / ivacaftor) in combination with ivacaftor (KALYDECO®) for the treatment of people with cystic fibrosis (CF) aged 12 years and older with certain mutations in the CFTR gene

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a favorable opinion for SYMKEVI® (tezacaftor / ivacaftor) in combination with ivacaftor (KALYDECO®) for the treatment of people with cystic fibrosis (CF) aged 12 years and older, who have either two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR), or a copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR protein exhibits residual activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711 + 3A → G, S945L, S977F, R1070W, D1152H, 2789 + 5G → A, 3272-26A → G, and 3849 + 10kbC → T.

If it receives a marketing authorization from the European Commission (EC), tezacaftor / ivacaftor will be used in combination with ivacaftor and will be the first drug to treat defects in the CFTR protein in CF patients with a copy of the F508del mutation and a copy of one of 14 mutations causing residual CTFR activity. It also provides a new therapeutic option for a significant number of people with CF who have two copies of the F508del mutation.

"At Vertex, our goal is to find a cure for all people living with CF. make rapid progress in treating up to 90 percent of patients, "says Dr. Reshma Kewalramani, Executive Vice President, Global Drug Development and Medical Affairs, and Medical Director at Vertex. "Today's announcement is a milestone in our mission. If approved, tezacaftor / ivacaftor in combination with ivacaftor represents a serious therapeutic option for people with two copies of the F508del mutation and the first drug in the EU for patients with a copy of the F508del mutation and a copy of one of the 14 mutations causing residual CTFR activity.

The regulatory submission was supported by the results of two pivotal Phase 3 studies, EVOLVE and EXPAND, published in the New England Journal of Medicine in November 2017. The results showed that tezacaftor / ivacaftor in combination with ivacaftor provides benefits on different CF populations, including statistically relevant improvements in lung function, as determined by the absolute baseline change in ppFEV1, and with a generally well-tolerated safety profile and no increase respiratory side effects compared with placebo. The most common adverse reactions experienced by patients receiving tezacaftor / ivacaftor in combination with ivacaftor in pooled, placebo-controlled Phase 3 studies were headache and nasopharyngitis.

"Tezacaftor combination therapy / ivacaftor for CF is another major step in the development of therapies that change the disease. This combination improves important clinical outcomes and could lead to improvements for people who can not use ORKAMBI® (lumacaftor / ivacaftor), "says Stuart Elborn, Professor of Respiratory Medicine, and Director of the Cystic Fibrosis Center at adult at Royal Brompton Hospital, London

Tezacaftor / ivacaftor in combination with ivacaftor was approved by the US Food and Drug Administration (FDA) in February 2018 and Health Canada in June 2018. It is marketed under the name SYMDEKO ™ in the United States and Canada

Source: Vertex