Vertex posts Phase 3 results for ELX/TEZ/IVA in CF infants 12 to 24 months

Vertex Pharmaceuticals posted results this week for a completed Phase 3 study of its triple combination cystic fibrosis therapy in young children.

• The trial, "A Phase 3, Open-label Study Evaluating the Pharmacokinetics, Safety, and Tolerability of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age" (NCT05882357), enrolled 67 participants in a non-randomized, sequential design study.¹
• In Part A, participants received ELX 40 milligram (mg)/TEZ 20 mg/IVA 30 mg fixed-dose combination granules once every morning and 25 mg IVA once every evening for 15 Days.¹ Measured Day 15 pre-dose concentrations ranged across six values, including 0.907 microgram per milliliter and 4.56 microgram per milliliter for different analytes.¹
• In Part A, 14 of 15 participants reported treatment-emergent adverse events, with no serious adverse events.¹
• Part B, which included some participants from Part A and newly enrolled participants,¹ used one of three weight-based dosing regimens, including ELX 80 mg/TEZ 40 mg/IVA 60 mg in the morning with 59.5 mg IVA in the evening for the highest dose group.¹
• In Part B, 53 of 54 participants had treatment-emergent adverse events and 6 had serious adverse events.¹
• The study, sponsored by Vertex Pharmaceuticals Incorporated, ran at 19 sites across 7 countries including Australia, Canada, Denmark, Germany, Netherlands, Switzerland, and the United Kingdom.¹