Orkambi wins CHMP’s thumbs-up for children ages 1 to 2

Medication is approved in EU to treat CF patients 2 and up

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Vertex Pharmaceuticals has received a positive opinion from an arm of the European Medicines Agency on expanding the use of Orkambi (lumacaftor/ivacaftor) to children ages 1-2 who have two copies of the F508del mutation, the most common genetic defect in cystic fibrosis (CF).

With the recommendation from the Committee for Medicinal Products for Human Use (CHMP), the company can expect the European Commission, which has authority to issue an approval for the European Union, to make a final decision soon.

“Today’s news provides great hope that, if approved, a treatment option will be available for the first time for about 300 young children with CF who have two copies of the F508del mutation,” Fosca De Iorio, vice president of international medical affairs at Vertex, said in a company press release.

CF is caused by mutations in the CFTR gene that makes mucus become thicker than normal and build up in the lungs, the gut, and other organs. The symptoms of CF begin to develop soon after birth and may vary in severity from person to person.

“Symptoms and organ damage start very early in the lives of people with [CF]. Treating patients as young as possible is important, as it can potentially slow the progression of this devastating disease,” De Iorio said.

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The CFTR gene provides instructions for making a protein channel of the same name, but the mutations can result in little to no CFTR protein on a cell’s surface or can affect the way the protein works.

Orkambi is an oral medication to treat CF. One of its active ingredients, lumacaftor, helps the CFTR protein form in the right shape so more of it reaches the cell’s surface. The other, ivacaftor, holds channels open, allowing the flow of chloride ions to be restored. Together, they prevent the buildup of thick and sticky mucus in the organs.

In the E.U., the medication is approved for people ages 2 and older who have two copies of the CF-causing F508del mutation. In the U.S., and more recently in Canada, its indication was expanded to cover children in their second year of life.

The expanded approval was supported by data from a company-sponsored Phase 3 clinical trial (NCT03601637) that tested Orkambi’s safety in children ages 1 to just younger than 2. The medication was generally safe and well tolerated, and reduced the sweat chloride concentration, a measure of the amount of salt in sweat that can tell how well the CFTR protein is working.