Last reviewed 2026-04-22 · How we verify

Ivacaftor/Ataluren

This combination therapy enhances defective CFTR protein function through two complementary mechanisms: ivacaftor acts as a CFTR potentiator to increase channel opening, while ataluren promotes read-through of nonsense mutations to restore full-length CFTR protein production.

This combination therapy enhances defective CFTR protein function through two complementary mechanisms: ivacaftor acts as a CFTR potentiator to increase channel opening, while ataluren promotes read-through of nonsense mutations to restore full-length CFTR protein production. Used for Cystic fibrosis with nonsense mutations (G542X and other nonsense CFTR mutations).

At a glance

Mechanism of action

Ivacaftor is a small-molecule potentiator that increases the open probability of CFTR channels at the cell surface, allowing more chloride ion transport. Ataluren is a nonsense suppression agent that enables ribosomes to read through premature stop codons, allowing production of full-length, functional CFTR protein in patients with nonsense mutations. Together, they address two distinct molecular defects in cystic fibrosis.

Approved indications

• Cystic fibrosis with nonsense mutations (G542X and other nonsense CFTR mutations)

Common side effects

• Respiratory tract infection
• Cough
• Headache
• Nausea

Key clinical trials

• PTC Study to Evaluate Ataluren in Combination With Ivacaftor (PHASE4)
• Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations (PHASE4)

Primary sources

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Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Ivacaftor/Ataluren CI brief — competitive landscape report
• Ivacaftor/Ataluren updates RSS · CI watch RSS
• University of Alabama at Birmingham portfolio CI