Cystic fibrosis: New treatment with three active ingredients

In a clinical study a combination of three drugs improved the lung function and quality of life in patients with cystic fibrosis significantly. If the therapy is used in children early on, might possibly even prevent the onset of the disease.

Compared to patients receiving a Placebo, improved lung function and quality of life through the triple combination of Elexacaftor, Tezacaftor and Ivacaftor significantly. The side effects were acceptable, it came in only one percent of the subjects in the termination of therapy.

"The results of the study suggest that with the new triple therapy in the future, up to 90 percent of all patients with cystic fibrosis can be treated. In professional circles this is considered a breakthrough in the treatment of this previously fatal Erbkrankheit" Prof. Dr. Marcus Mall, Director of the Christiane Herzog cystic fibrosis centre at the Charité hospital explains.

Triple combination in the United States already admitted

Through the combination of active ingredients with different functions are affected likely to be affected by the genetic disease. Thus, the onset of the disease could prevent as early as infancy, or at least delay the leave. In the US the drug has been approved in a fast method already, and in Europe, the corresponding applications were submitted to the competent authorities.

100 years ago, you could not help children suffering from cystic fibrosis. Most died before they reached adulthood. By a disturbed salt and water transport in the mucous membranes, the secretions are viscous, which is particularly in the respiratory tract is noticeable. About 90 percent of the cases are caused by a Mutation, against the tested combination of active ingredients helps.