Ivacaftor/Ataluren — Competitive Intelligence Brief

Ivacaftor/Ataluren (Ivacaftor/Ataluren) competitive landscape: 6 comparators, recent regulatory actions, upcoming PDUFA, patent timeline. Class: CFTR modulator combination. Area: Respiratory / Genetic Disorders.

marketed CFTR modulator combination CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Respiratory / Genetic Disorders Small molecule

Target snapshot

Ivacaftor/Ataluren (Ivacaftor/Ataluren) — University of Alabama at Birmingham. 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.

Comparator set (6 drugs)

Drug	Generic	Sponsor	Phase	Class	Target	Approval / PDUFA
Ivacaftor/Ataluren TARGET	Ivacaftor/Ataluren	University of Alabama at Birmingham	marketed	CFTR modulator combination	CFTR (Cystic Fibrosis Transmembrane Conductance Regulator)
LUM/IVA	LUM/IVA	Vertex Pharmaceuticals Incorporated	marketed	CFTR modulator combination (corrector + potentiator)	CFTR (cystic fibrosis transmembrane conductance regulator)
Ivacaftor+lumacaftor	Ivacaftor+lumacaftor	Assistance Publique - Hôpitaux de Paris	marketed	CFTR modulator combination (corrector + potentiator)	CFTR (cystic fibrosis transmembrane conductance regulator)
Elexacaftor / Ivacaftor / Tezacaftor	Elexacaftor / Ivacaftor / Tezacaftor	Nottingham University Hospitals NHS Trust	marketed	CFTR modulator combination (correctors + potentiator)	CFTR (cystic fibrosis transmembrane conductance regulator)
Lumacaftor plus Ivacaftor	Lumacaftor plus Ivacaftor	Hannover Medical School	phase 3	CFTR modulator combination (corrector + potentiator)	CFTR (cystic fibrosis transmembrane conductance regulator)
ELX/TEZ/IVA	ELX/TEZ/IVA	Vertex Pharmaceuticals Incorporated	phase 3	CFTR modulator combination	CFTR (cystic fibrosis transmembrane conductance regulator)
Placebo matched to VX-661/ ivacaftor	Placebo matched to VX-661/ ivacaftor	Vertex Pharmaceuticals Incorporated	phase 3	CFTR modulator combination (corrector + potentiator)	CFTR (Cystic Fibrosis Transmembrane Conductance Regulator)

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this set.

Patent timeline (forward window)

No upcoming patent expiries for this set.

Sponsor landscape (CFTR modulator combination class)

1. University of Alabama at Birmingham · 1 drug in this class
2. Vertex Pharmaceuticals Incorporated · 1 drug in this class