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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
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.
Vertex Pharmaceuticals Inc.
50 Northern Avenue
Boston, Massachusetts 02210
United States

The sponsor address listed is the last reported by the sponsor to OOPD.
 
Generic Name:  elexacaftor/tezacaftor/ivacaftor
Trade Name:  TRIKAFTA
Date Designated:  08/29/2018
Orphan Designation:  Treatment of Cystic Fibrosis
Orphan Designation Status:  Designated/Approved
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.
Vertex Pharmaceuticals Inc.
50 Northern Avenue
Boston, Massachusetts 02210
United States

The sponsor address listed is the last reported by the sponsor to OOPD.
 
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
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
Vertex Pharmaceuticals Inc.
50 Northern Avenue
Boston, Massachusetts 02210
United States

The sponsor address listed is the last reported by the sponsor to OOPD.
*Exclusivity Protected Indications are shown for approvals from Jan. 1, 2013, to the present.
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