EU’s CHMP Backs Kalydeco, Symkevi License Extension for Use in Younger Children

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by Marisa Wexler MS |

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Kalydeco, Symkevi License Extension

The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended extending the licenses for Symkevi and Kalydeco, two cystic fibrosis (CF) treatments marketed by Vertex Pharmaceuticals, to allow these medications to be used in younger children.

If approved by the European Commission, these extensions would allow the use of Symkevi (tezacaftor/ivacaftor combo, known as Symdeko in the U.S.) to treat children with CF as young as age 6, and the administration of Kalydeco in infants as young as 4 months old.

“Today’s recommendations to license Symkevi for children aged 6 to 11 and Kalydeco for babies aged four months to six months are a significant step forward in CF care, and pave the way for thousands of children to have access to a new disease-modifying treatment option,” Keith Brownlee, director of policy, programs, and support at the Cystic Fibrosis Trust, in the U.K., said in a press release.

“The next step is for the European Commission to formally issue the licences, which we expect to take around two months. After this, parents will be able to speak to their CF team for more details on their child’s eligibility and getting access to these drugs,” Brownlee added.

CF is caused by mutations in the CFTR gene, which leads to defects in the CFTR protein ultimately resulting in the symptoms of the disease.

Ivacaftor, the active ingredient in Kalydeco, is a CFTR modulator — a type of therapy that works directly on the defective CFTR protein, allowing the protein to function more effectively. Symkevi also contains ivacaftor, in combination with another CFTR modulator called tezacaftor.

Currently in the EU, Kalydeco’s approval covers the medication’s use in children as young as six months, while the approval for Symkevi allows its use in patients age 12 or older.

Because these medications work on particular defects in the CFTR protein, they are only indicated for people with specific underlying CFTR mutations. Symkevi is indicated for people with two copies of the F508del mutation — the most common CF-causing mutation — or one F508del and a residual function mutation. Kalydeco is indicated for young children with one of 38 specific mutations.

Vertex previously announced positive results with Symkevi in a Phase 3 clinical trial (NCT03559062) that enrolled 67 children with CF from 6 to 11 years old. The results showed that the treatment significantly improved lung function and was generally safe and well-tolerated.


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