FDA expands approval of Orkambi for young children with cystic fibrosis
Click Here to Manage Email Alerts
The FDA expanded the approval of lumacaftor and ivacaftor to include the treatment of children with cystic fibrosis aged 1 year to younger than 2 years, according to a manufacturer-issued press release.
This approval applies to children who are homozygous for the F508del mutation (F/F genotype) in the CFTR gene.
Harboring two copies of the F508del mutation leads to a deficiency of the CFTR protein at the cell surface. The agent combines lumacaftor, which targets the defect of the F508del-CFTR protein that inhibits processing and trafficking, thereby increasing the amount of the protein at the cell surface; and ivacaftor, a CFTR potentiator, which facilitates the ability of CFTR proteins to transport salt and water across the cell membrane. Together, these two actions help hydrate and clear mucus from the airways.
Lumacaftor and ivacaftor (Orkambi, Vertex Pharmaceuticals) was originally approved in 2015 for children with cystic fibrosis aged 12 years and older, with later approvals extending it to younger children.
The current expanded approval was based, in part, on data from a 24-week, phase 3, open-label multicenter study that included 46 children aged 1 year to younger than 2 years with cystic fibrosis and the F/F genotype.
Results showed lumacaftor and ivacaftor was well tolerated, with similar safety and pharmacokinetics as observed among older children. Data also showed that the treatment led to a reduction in sweat chloride concentration, suggesting the possibility of cystic fibrosis disease modification.
“Treating children with cystic fibrosis as early in life as possible is critically important, because early treatment has the potential to slow the progression of this devastating disease,” Carmen Bozic, MD, executive vice president of global medicines development and medical affairs and chief medical officer of Vertex, said in the release. “[This] approval is another important step on our journey to reach people of all ages living with cystic fibrosis who may benefit from our medicines.”