EMA, the European Medicines Agency, approved Kaftrio, a new triple-combination therapy for the treatment of cystic fibrosis on August 21. Approval has been given for patients ages 12 years and older, who are either homozygous for the F508del mutation that most fre-quently causes cystic fibrosis or have a F508del mutation in combination with a minimal function mutation. Currently, approximately 60 percent of cystic fibrosis patients in Ger-many are expected to benefit from this new drug.
The FDA (Food and Drug Administration) reported in October 2019 that the combination of three substances was “groundbreaking”. The drug was approved by the American authority in less than eight weeks. The European approval for the triple-combination therapy is not quite as comprehensive as that for Trikafta, its U.S. equivalent. Following the current ap-proval, approx. 60 percent of people affected by cystic fibrosis in Germany can now benefit from the new drug. This number is based on analyses from the German Cystic Fibrosis Regis-try and expected to gradually increase to about 85 percent within the years to come once Kaftrio approval is expanded to include further age groups and indications.
Professor Dr. Marcus Mall, Medical Director, Head of Department of Pediatrics, Division of Pneumonology, Immunology and Intensive Care at the Charité Berlin, professor at the Berlin Institute of Health (BIH), and long-standing DZL board member, played a vital role in the clinical trial of the drug. “Kaftrio probably means a complete change in the therapy of cystic fibrosis,” he explains. Together with Privatdozent Dr. Mirjam Stahl, Mall is currently in the process of preparing the German part of international testing of the new drug in younger children. He estimates that, as soon as in five years, the preventive treatment of infants with inhalation therapy could be replaced by the drug whose efficacy has recently been shown for the first time by him and Stahl in the multi-centric DZL study PRESIS (Preventive Inhalation of Hypertonic Saline in Infants with Cystic Fibrosis).
However, there is still one group of patients for which Kaftrio is not suitable – due to mutations, transplantations or other severe illnesses, such as liver or kidney disorders. Further innovative research will be required for these patients in the future.
Cystic fibrosis (Mucoviscidosis) is the most common hereditary lung disease occurring at a young age and is still terminal. In Germany, approximately 1:2,500 newborns is affected. Thanks to the progress in symptomatic treatment options and due to standardized medical care, the median survival age of patients in Germany has already increased to about 40 years. Currently, and despite significant progress in the development of new drugs for the pharmacological correction of genetically modified CFTR chloride channels, for most patients, there is still no therapy that effectively fights the cause of cystic fibrosis. Consequently, objective of the cystic fibrosis research program is to obtain a better understanding of the pathogenesis and improve diagnostics as well as develop new strategies for the effective prevention and treatment of cystic fibrosis.