Orkambi Approved in Europe for Younger CF Patients with F508del Mutations

Alice Melão, MSc avatar

by Alice Melão, MSc |

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The European Commission has approved the label extension of Orkambi (lumacaftor/ivacaftor) for the treatment of children ages 2-5 with cystic fibrosis (CF) due to F508del mutations, Vertex Pharmaceuticals, the therapy’s manufacturer, announced.

Orkambi was previously approved in Europe to treat CF patients 6 and older with the F508del mutation in the CFTR gene, the most common genetic defect associated with CF. This latest approval extends the coverage of the therapy for up to 1,500 children whose CF is caused by two copies of the F508del mutation.

“[The] approval by the European Commission brings us one step closer to our goal of bringing treatment to all people living with CF,” Reshma Kewalramani, MD, executive vice president of global medicines development and medical affairs and chief medical officer at Vertex, said in a press release. “By treating the underlying cause of disease early, we can potentially modify its course and offer patients the chance of improved outcomes.”

Vertex’s request to expand Orkambi’s marketing authorization in Europe was based on data from a Phase 3 open-label trial (NCT03125395), and was supported by the positive recommendation of the European Committee for Medicinal Products for Human Use (CHMP).

The clinical trial evaluated Orkambi’s safety in about 60 CF patients age 2 and older who had two copies of the F508del mutation. The children received the treatment every 12 hours, according to their age and weight.

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Clinical data showed that treatment with Orkambi for 24 weeks was well-tolerated and generally safe, with children ages 2-5 showing a similar safety profile to those 6 and older. The most common adverse event reported was cough, affecting 63% of the children in the group. Still, most of the adverse events reported were mild or moderate in severity.

Efficacy data at week 24 also demonstrated that Orkambi could effectively reduce the levels of sweat chloride — a hallmark of the disease — in these children. Improvements in growth parameters, such as weight and height, were also reported.

In August 2018, the U.S. Food and Drug Administration extended the therapy’s label for use in children ages 2-5. Health Canada approved Orkambi’s label extension in December 2018.