Phase 3 Results from Two Studies of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Triple Combination Treatment for Cystic Fibrosis Concurrently Published in The New England Journal of Medicine and The Lancet - Seite 2

The results were published online in conjunction with the presentation of both studies at the 33rd Annual North American Cystic Fibrosis Conference (NACFC), October 31 through November 2 in Nashville. The oral presentation highlighting key outcomes from both studies is scheduled on Friday, November 1 at 2:15 p.m. CT.

These studies form the basis of the recent approval of TRIKAFTA by the U.S. FDA and support the Marketing Authorization Application (MAA) currently under review with the European Medicines Agency. Other global submissions are also being prepared and will be submitted in 2020.

Additional presentations at NACFC highlight data from across Vertex’s CF portfolio, including 96-week long-term safety and efficacy data for SYMDEKO (tezacaftor/ivacaftor and ivacaftor) in patients ages >12 years with CF homozygous for F508del-CFTR (F/F) or heterozygous for F508del-CFTR and a residual function mutation (F/RF), as well as data demonstrating the burden of illness in F/MF patients >12 years of age.

	Abstract Title	Presentation Type	Presenting Author	Date/Time
ELX/TEZ/IVA	Phase 3 efficacy and safety of the ELX/TEZ/IVA triple combination in people with CF homozygous for the F508del mutation	Poster #508	Harry Heijerman	Thursday, October 31 11:15 a.m. to 1:45 p.m.
	Phase 3 efficacy and safety of the ELX/TEZ/IVA triple combination in people with CF and F508del/minimal function genotypes	Poster #507	Raksha Jain	Thursday, October 31 11:15 a.m. to 1:45 p.m.
	Phase 3 efficacy and safety of the ELX/TEZ/IVA combination therapy in people with CF homozygous for F508del or heterozygous for F508del and a minimal function mutation	Oral Presentation: Workshop 17	Raksha Jain	Friday, November 1 2:15 p.m. to 3:45 p.m.
TEZ / IVA