EMA committee supports Kalydeco for children as young as 1 month

Approval would make therapy first CFTR modulator for this patient population

Steve Bryson, PhD avatar

by Steve Bryson, PhD |

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A European Medicines Agency (EMA) committee recommends extending the label for Kalydeco (ivacaftor) to children with cystic fibrosis (CF) as young as a month who carry at least one CFTR gating mutation.

If the European Commission (EC) accepts the endorsement by the Committee for Medicinal Products for Human Use (CHMP), an arm of the EMA, Kalydeco would be available to most CF patients in Europe. Historically, the EC accepts most CHMP opinions.

“Cystic fibrosis is a chronic, progressive disease that is present from birth, which is why it is important to treat the underlying cause of this disease as early as possible to potentially slow disease progression,” Fosca De Iorio, vice president of international medical affairs at Vertex Pharmaceuticals, the company that markets the therapy, said in a company press release.

In CF, inherited mutations in the CFTR gene lead to a missing or defective CFTR protein, which acts like a gate to regulate the flow of chloride into and out of cells. Mutations in the CFTR gene lead to abnormally thick mucus building up in various organs. Gating mutations, in particular, impair the gate-like function of the CFTR protein, causing it to become stuck closed.

A part of the CFTR modulator class of medicines, Kalydeco is known as a CFTR potentiator because it enhances the activity of the CFTR protein by keeping the gate open longer at the cell surface. This improves the flow of water and salt, reducing the thickness of mucus.

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Who would Kalydeco’s extension apply to?

Kalydeco was first approved for patients 2 and older in Europe, then extended in 2018 to treat 1-year-olds. The EC eventually extended its label to infants as young as 6 months and later as young as 4 months who have CF caused by certain mutations.

The latest extension would apply to European children as young as a month who carry CFTR gating mutations R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.

The approval would make Kalydeco the first and only CFTR modulator approved for this patient population. In the U.S., the CFTR potentiator is also available to month-old infants.

The approvals were supported by data from the completed Phase 3 ARRIVAL trial (NCT02725567). The therapy’s safety, pharmacological properties, and preliminary efficacy were evaluated in CF patients younger than 24 months (2 years). Its safety profile was similar to that reported in older children and adults, according to Vertex.

“If the label expansion for Kalydeco is approved, this would allow eligible infants with CF to receive a treatment that targets the underlying cause of their disease at the very start of their lives,” De Iorio said.