Cystic fibrosis therapy that improves cell function granted EU approval

The drug, Symkevi (tezacaftor/ivacaftor) was given marketing authorization by the European Commission (EC) as a therapy for cystic fibrosis (CF) patients with two copies of the F508del mutation, and a single copy of one of the 14 mutations that causes protein channel activity.

It becomes the first treatment available in the European Union (EU) for this specific condition; it also offers another treatment option for patients with two copies of the f508del mutation, the most common mutation in cystic fibrosis.

A Vertex spokesperson told us, “A mutation in the genes of a person with CF may result in proteins that don’t make it to the cell surface, where they are normally located, or in proteins at the cell surface that doesn’t open enough.” ​

The EC’s marketing authorization was based on results from two Phase III studies. The studies showed treatment with Symkevi provided benefits across different CF populations – statistically, significant improvements in lung function were also seen.

The treatment was approved by the U.S. Food and Drug Administration in February 2018 and by Health Canada in June 2018.

“While treatment is advancing rapidly, many people living with CF still do not have a therapy that targets the cause of their form of the disease,”​ explained the spokesperson.

With this authorization though, targeted treatment for CF is another step closer, she added.

Symkevi combines active agents tezacaftor with ivacaftor, also known by the brand name Kalydeco – when combined they help improve the performance of the CFTR channel in CF patients.

Tezacaftor helps the CFTR protein onto the cell surface and ivacaftor then helps the CFTR ion channel to remain open for longer periods. Together the active agents improve the balance of water and salts in affected organs.