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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: tezacaftor/ivacaftor; ivacaftor
Trade Name: Symdeko
Date Designated: 06/15/2017
Orphan Designation: Treatment of cystic fibrosis (CF)
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: tezacaftor/ivacaftor; ivacaftor
Trade Name: Symdeko
Marketing Approval Date: 02/12/2018
Approved Labeled Indication: SYMDEKO a combination of tezacaftor and ivacaftor, is indicated for the treatment of patients with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence
Exclusivity End Date: 02/12/2025 
Exclusivity Protected Indication* :  SYMDEKO a combination of tezacaftor and ivacaftor, is indicated for the treatment of patients with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence
2 Generic Name: tezacaftor/ivacaftor; ivacaftor
Trade Name: Symdeko
Marketing Approval Date: 06/21/2019
Approved Labeled Indication: SYMDEKO? is indicated for the treatment of patients with cystic fibrosis (CF) age 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.
Exclusivity End Date: 06/21/2026 
Exclusivity Protected Indication* :  For the treatment of patients with cystic fibrosis (CF) age 6 years to less than 12 years who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence
3 Generic Name: tezacaftor/ivacaftor; ivacaftor
Trade Name: Symdeko
Marketing Approval Date: 12/21/2020
Approved Labeled Indication: treatment of cystic fibrosis (CF) in patients age 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence
Exclusivity End Date: 12/21/2027 
Exclusivity Protected Indication* :  treatment of cystic fibrosis (CF) in patients age 6 years and older who have at least one of the additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that have been identified as responsive to tezacaftor/ivacaftor based on in vitro data and identified in the approval on December 21, 2020
*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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