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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Generic Name:  ivacaftor
Trade Name:  Kalydeco
Date Designated:  12/20/2006
Orphan Designation:  Treatment of patients with cystic fibrosis
Orphan Designation Status:  Designated/Approved
Marketing Approval Date:  01/31/2012 
Approved Labeled Indication:  Treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR gene.
Exclusivity End Date:    01/31/2019 
Exclusivity Protected Indication* :  
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:  ivacaftor
Trade Name:  Kalydeco
Date Designated:  12/20/2006
Orphan Designation:  Treatment of patients with cystic fibrosis
Orphan Designation Status:  Designated/Approved
Marketing Approval Date:  04/29/2019 
Approved Labeled Indication:  KALYDECO is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients age 6 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data
Exclusivity End Date:    04/29/2026 
Exclusivity Protected Indication* :  For the treatment of cystic fibrosis (CF) in patients age 6 months to less than 12 months who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay 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:  ivacaftor
Trade Name:  Kalydeco
Date Designated:  12/20/2006
Orphan Designation:  Treatment of patients with cystic fibrosis
Orphan Designation Status:  Designated/Approved
Marketing Approval Date:  02/21/2014 
Approved Labeled Indication:  Treatment of cystic fibrosis (CF) in patients age 6 years and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.
Exclusivity End Date:    02/21/2021 
Exclusivity Protected Indication* :  Treatment of cystic fibrosis (CF) in patients age 6 years and older who have one of the following mutations in the CFTR gene: G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.
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:  ivacaftor
Trade Name:  Kalydeco
Date Designated:  12/20/2006
Orphan Designation:  Treatment of patients with cystic fibrosis
Orphan Designation Status:  Designated/Approved
Marketing Approval Date:  12/29/2014 
Approved Labeled Indication:  Treatment of cystic fibrosis (CF) in patients age 6 years and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R; and treatment of CF in patients age 6 years and older who have an R117H mutation in the CFTR gene.
Exclusivity End Date:    12/29/2021 
Exclusivity Protected Indication* :  Treatment of cystic fibrosis (CF) in patients age 6 years and older who have an R117H mutation in the 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:  ivacaftor
Trade Name:  Kalydeco
Date Designated:  12/20/2006
Orphan Designation:  Treatment of patients with cystic fibrosis
Orphan Designation Status:  Designated/Approved
Marketing Approval Date:  03/17/2015 
Approved Labeled Indication:  Treatment of cystic fibrosis (CF) in patients age 2 years and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R ; and treatment of CF in patients age 2 years and older who have an R117H mutation in the CFTR gene.
Exclusivity End Date:    03/17/2022 
Exclusivity Protected Indication* :  Treatment of cystic fibrosis (CF) in patients ages 2 to less than 6 years who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, and R117H.
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:  ivacaftor
Trade Name:  Kalydeco
Date Designated:  12/20/2006
Orphan Designation:  Treatment of patients with cystic fibrosis
Orphan Designation Status:  Designated/Approved
Marketing Approval Date:  05/17/2017 
Approved Labeled Indication:  Treatment of cystic fibrosis (CF) in patients age 2 years and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.
Exclusivity End Date:    05/17/2024 
Exclusivity Protected Indication* :  Treatment of cystic fibrosis (CF) in patients age 2 years and older who have one of the following mutations in the CFTR gene: E56K, P67L, R74W, D110E, D110H, R117C, E193K, L206W, R347H, R352Q, A455E, D579G, S945L, S977F, F1052V, K1060T, A1067T, G1069R, R1070Q, R1070W, F1074L, D1152H, and D1270N.
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:  ivacaftor
Trade Name:  Kalydeco
Date Designated:  12/20/2006
Orphan Designation:  Treatment of patients with cystic fibrosis
Orphan Designation Status:  Designated/Approved
Marketing Approval Date:  07/31/2017 
Approved Labeled Indication:  Treatment of cystic fibrosis (CF) in patients age 2 years and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.
Exclusivity End Date:    07/31/2024 
Exclusivity Protected Indication* :  Treatment of cystic fibrosis (CF) in patients age 2 years and older who have one of the following mutations in the CFTR gene: 711+3A G, E831X, 2789+5G A, 3272-26A G, and 3849+10kbC T.
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:  ivacaftor
Trade Name:  Kalydeco
Date Designated:  12/20/2006
Orphan Designation:  Treatment of patients with cystic fibrosis
Orphan Designation Status:  Designated/Approved
Marketing Approval Date:  08/15/2018 
Approved Labeled Indication:  Treatment of cystic fibrosis (CF) in patients age 12 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.
Exclusivity End Date:    08/15/2025 
Exclusivity Protected Indication* :  For the treatment of cystic fibrosis (CF) in patients age 12 months to less than 2 years who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay 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:  ivacaftor
Trade Name:  Kalydeco
Date Designated:  12/20/2006
Orphan Designation:  Treatment of patients with cystic fibrosis
Orphan Designation Status:  Designated/Approved
Marketing Approval Date:  12/21/2020 
Approved Labeled Indication:  treatment of cystic fibrosis (CF) in patients age 4 months and older who have one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data
Exclusivity End Date:    12/21/2027 
Exclusivity Protected Indication* :  treatment of cystic fibrosis (CF) in patients age 4 months and older who have one of the additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that have been identified as responsive to ivacaftor potentiation based on in vitro data and identified in the approval on December 21, 2020.
Vertex Pharmaceuticals Inc.
50 Northern Ave
Boston, Massachusetts 02210
United States

The sponsor address listed is the last reported by the sponsor to OOPD.
 
Generic Name:  ivacaftor
Trade Name:  KALYDECO®
Date Designated:  12/20/2006
Orphan Designation:  Treatment of patients with cystic fibrosis
Orphan Designation Status:  Designated/Approved
Marketing Approval Date:  09/24/2020 
Approved Labeled Indication:  KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients age 4 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data
Exclusivity End Date:    09/24/2027 
Exclusivity Protected Indication* :  treatment of cystic fibrosis (CF) in patients age 4 months to less than 6 months who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay 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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