Alyftrek is an innovative therapy that targets the underlying cause of cystic fibrosis (CF). The European Medicines Agency (EMA) has granted approval for patients ≥12 years with non–class I mutations. This decision marks a significant victory for patients with CF. In clinical trials, Alyftrek was as effective as Kaftrio, a gold-standard triple combination therapy that has already been approved.
This approval comes after a successful run of two randomized, phase 3, double-blinded clinical studies on CF patients. These studies reinforced that Alyftrek is just as effective as Kaftrio, which is made up of elexacaftor, tezacaftor and ivacaftor. Both unprecedented medications serve the same patient population, giving hope to those impacted by this genetic condition.
The clinical trials evaluating Alyftrek documented changes in lung function in terms of percent predicted forced expiratory volume in 1 second (FEV1). After 24 weeks of treatment, patients showed significant improvements in their lung function. In another randomized, double-blind, placebo-controlled trial, Alyftrek was found to notably delay the progression of the disease over a two-year period. This delay was quantified with the Neuropathy Impairment Score with an addition of 7 nerve tests (NIS + 7). The results were encouraging, providing a significant delay in disease progression relative to placebo.
Alyftrek was granted orphan drug status for the treatment of cystic fibrosis in the European Union in 2021. This designation further demonstrates the critical need to advance new therapies for rare diseases that impact only a small population of patients. Alyftrek is available as film-coated tablets. Flexible dosage options, including 4 mg, 75 mg, and 125 mg offer the opportunity for tailored treatment regimens that meet the individual needs of patients.
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