Benefits of long-term and early treatment with CFTR modulators

Five scientific abstracts on Vertex's cystic fibrosis drugs were presented at the 45th Conference of the European Cystic Fibrosis Society (ECFS) held in Rotterdam, the Netherlands, on June 8-11, 2022.

The presented analysis of data collected in the US CF Foundation Patient Registry CFFPR covering more than
16 thousand in subjects treated with ivacaftor/tezacaftor/elexacaftor in the combination regimen with ivacaftor for an average of nine months, showed that such treatment under real conditions was associated with an improvement in lung function, a reduction in the risk of lung disease exacerbations by 77% compared to the pre-treatment period , as well as a 87% reduction in the risk of lung transplantation and a 74% reduction in the risk of death, compared to the historical 2019 data of the population
from the US CFFPR register. No new side effects were identified.

Vertex also provided data comparing the annual rate of change in lung function
in subjects 12 years of age and older with cystic fibrosis with two F508del (F/F) or one F508del and one minimal function (F/MF) mutation treated with ivacaftor/tezacaftor/elexacaftor in a combination regimen with ivacaftor in the pivotal and open-label studies the extension study, compared with historical propensity score matching (PMS) statistical controls that did not use CFTR modulators and were derived from the US CFFPR registry (WS22.04 abstract). The results show that patients receiving ivacaftor/tezacaftor/elexacaftor in the ivacaftor combination regimen did not experience a decrease in ppFEV1 over a two-year period, contrary to the decreases seen in the matched controls. The analysis shows that the studied therapy has an influence on the course of lung disease caused by cystic fibrosis.

In addition, Vertex has presented data from a long-term study in routine clinical practice (RWS) showing that starting ivacaftor early in life (6-10 years of age) preserves lung function
to a greater extent than in the case of starting the drug in an older age (abstract WS17.03). The results of the study support the importance of early initiation of treatment in eligible patients.

In addition to the research mentioned above, other Vertex presentations at this year's ECFS conference confirm the importance of long-term and early use of CFTR modulators:

• Abstract WS08.04 - Results of a study in routine clinical practice (RWS) in cystic fibrosis patients with selected residual function mutations treated with ivacaftor.
• Abstract WS17.02 - ORKAMBI ^® (lumacaftor/ivacaftor) Phase 2 open-label extension study results in children with cystic fibrosis aged 2-5 years.

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