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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: elexacaftor, tezacaftor, and ivacaftor; ivacaftor
Trade Name: Trikafta
Date Designated: 08/29/2018
Orphan Designation: Treatment of Cystic Fibrosis
Orphan Designation Status: Designated/Approved
Vertex Pharmaceuticals Incorporated
50 Northern Avenue
Boston, Massachusetts 02210
United States

The sponsor address listed is the last reported by the sponsor to OOPD.

Marketing approved:

1 Generic Name: elexacaftor, tezacaftor, and ivacaftor; ivacaftor
Trade Name: Trikafta
Marketing Approval Date: 10/21/2019
Approved Labeled Indication: TRIKAFTA is indicated for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
Exclusivity End Date: 10/21/2026 
Exclusivity Protected Indication* :  Indicated for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
2 Generic Name: elexacaftor, tezacaftor, and ivacaftor; ivacaftor
Trade Name: Trikafta
Marketing Approval Date: 12/21/2020
Approved Labeled Indication: treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
Exclusivity End Date: 12/21/2027 
Exclusivity Protected Indication* :  treatment of cystic fibrosis in patients aged 12 years and older who have a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive based on in vitro data.
3 Generic Name: elexacaftor, tezacaftor, and ivacaftor; ivacaftor
Trade Name: Trikafta
Marketing Approval Date: 06/08/2021
Approved Labeled Indication: Treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
Exclusivity End Date: 06/08/2028 
Exclusivity Protected Indication* :  Treatment of cystic fibrosis (CF) in patients aged 6 through 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
4 Generic Name: elexacaftor, tezacaftor, and ivacaftor; ivacaftor
Trade Name: Trikafta
Marketing Approval Date: 04/26/2023
Approved Labeled Indication: treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
Exclusivity End Date: 04/26/2030 
Exclusivity Protected Indication* :  treatment of cystic fibrosis (CF) in patients aged 2 years to less than 6 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data
5 Generic Name: elexacaftor, tezacaftor, and ivacaftor; ivacaftor
Trade Name: Trikafta
Marketing Approval Date: 12/20/2024
Approved Labeled Indication: treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on clinical and/or in vitro data
Exclusivity End Date: 12/20/2031 
Exclusivity Protected Indication* :  treatment of cystic fibrosis (CF) in patients aged 2 years and older who have one of the additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive based on clinical and/or in vitro data and identified in the approval on December 20, 2024
6 Generic Name: elexacaftor, tezacaftor, and ivacaftor; ivacaftor
Trade Name: Trikafta
Marketing Approval Date: 03/27/2026
Approved Labeled Indication: treatment of cystic fibrosis (CF) in adult and pediatric patients aged 2 years and older who have a clinical diagnosis of CF and who have at least one variant in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein
Exclusivity End Date: TBD  
*Exclusivity Protected Indications are shown for approvals from 01/01/2013 to the present.
*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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