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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
Vertex Pharmaceuticals, Inc.
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: ivacaftor
Trade Name: Kalydeco
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* :  
2 Generic Name: ivacaftor
Trade Name: Kalydeco
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.
3 Generic Name: ivacaftor
Trade Name: Kalydeco
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.
4 Generic Name: ivacaftor
Trade Name: Kalydeco
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.
5 Generic Name: ivacaftor
Trade Name: Kalydeco
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.
6 Generic Name: ivacaftor
Trade Name: Kalydeco
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.
7 Generic Name: ivacaftor
Trade Name: Kalydeco
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
8 Generic Name: ivacaftor
Trade Name: Kalydeco
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
9 Generic Name: ivacaftor
Trade Name: Kalydeco
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
10 Generic Name: ivacaftor
Trade Name: Kalydeco
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.
11 Generic Name: ivacaftor
Trade Name: Kalydeco
Marketing Approval Date: 05/03/2023
Approved Labeled Indication: treatment of cystic fibrosis (CF) in patients age 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data
Exclusivity End Date: 05/03/2030 
Exclusivity Protected Indication* :  treatment of cystic fibrosis (CF) in patients 1 month to less than 4 months of age who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data
*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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