dm+d

Unassigned

New Medicines

Cystic fibrosis - as a triple combination with VX-121 and tezacaftor for certain mutations in the CFTR gene

Information

New formulation
Vertex
Vertex

Development and Regulatory status

None
None
Phase III Clinical Trials
Yes

Category

Deuterated ivacaftor has been developed by replacing specific hydrogen atoms with deuterium atoms. Selective replacement of hydrogen with deuterium allows retention of full potency and selectivity of the original, but with improved pharmacokinetics [1].
Cystic fibrosis (CF) affects approximately 75,000 people in North America, Europe and Australia and is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. There are approximately 2,000 known mutations in the CFTR gene. The median age of death is in the mid-to-late 20s [2].
Cystic fibrosis - as a triple combination with VX-121 and tezacaftor for certain mutations in the CFTR gene
Oral