Efficacy

Efficacy of Symdeko (tezacaftor/ivacaftor) in Cystic Fibrosis

Symdeko, which is a combination of tezacaftor and ivacaftor, is a medication approved for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have two copies of the F508del mutation in the CFTR gene or at least one mutation that is responsive to tezacaftor/ivacaftor. The efficacy of Symdeko in cystic fibrosis is primarily measured by improvements in lung function, reduction in pulmonary exacerbations, and enhancements in the quality of life for patients. Clinical trials have demonstrated that Symdeko can lead to significant improvements in the percent predicted forced expiratory volume in one second (ppFEV1), which is a key indicator of lung function in CF patients.

In the pivotal phase 3 clinical trials, known as the EVOLVE and EXPAND studies, Symdeko showed a statistically significant improvement in lung function compared to placebo. Participants treated with Symdeko experienced fewer pulmonary exacerbations, which are episodes of worsening respiratory symptoms that often require additional treatment and can lead to a decline in lung function over time. Additionally, patients reported improvements in respiratory symptoms and overall health status, as indicated by the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score.

Efficacy of Symkevi (tezacaftor/ivacaftor) in Cystic Fibrosis

Symkevi, known as Symdeko in the United States, is the combination of tezacaftor and ivacaftor used for the treatment of cystic fibrosis in patients with certain mutations in the CFTR gene. In the European Union and other regions, Symkevi has been approved for use in patients aged 12 years and older with either two copies of the F508del mutation or one F508del mutation and one mutation that results in a CFTR protein responsive to the combination therapy. The efficacy of Symkevi in improving lung function and reducing exacerbations has been demonstrated in clinical trials similar to those of Symdeko, with comparable outcomes in terms of ppFEV1 improvements and reductions in the rate of pulmonary exacerbations.

The safety and efficacy of Symkevi were evaluated in a clinical development program that included patients with the relevant CFTR mutations. The improvements in lung function and the quality of life, along with the reduction in exacerbations, support the use of Symkevi as a treatment option for eligible cystic fibrosis patients. It is important to note that the response to Symkevi may vary based on individual patient factors, including genotype and baseline lung function. Symkevi, like Symdeko, is part of a class of drugs known as CFTR modulators, which aim to correct the function of the defective protein made by the CFTR gene.