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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: lumacaftor/ivacaftor
Trade Name: Orkambi
Date Designated: 06/30/2014
Orphan Designation: Treatment of 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: lumacaftor/ivacaftor
Trade Name: Orkambi
Marketing Approval Date: 07/02/2015
Approved Labeled Indication: Treatment of cystic fibrosis in patients age 12 years and older who are homozygous for F508del mutation in the CFTR gene
Exclusivity End Date: 07/02/2022 
Exclusivity Protected Indication* :  Treatment of cystic fibrosis in patients age 12 years and older who are homozygous for F508del mutation in the CFTR gene
2 Generic Name: lumacaftor/ivacaftor
Trade Name: Orkambi
Marketing Approval Date: 09/28/2016
Approved Labeled Indication: Treatment of cystic fibrosis (CF) in patients age 6 years and older who are homozygous for the F508del mutation in the CFTR gene
Exclusivity End Date: 09/28/2023 
Exclusivity Protected Indication* :  Treatment of cystic fibrosis (CF) in patients age 6-11 years old who are homozygous for the F508del mutation in the CFTR gene
3 Generic Name: lumacaftor/ivacaftor
Trade Name: Orkambi
Marketing Approval Date: 08/07/2018
Approved Labeled Indication: ORKAMBI is a combination of lumacaftor and ivacaftor indicated for the treatment of cystic fibrosis (CF) in patients age 2 years and older who are homozygous for the F508del mutation in the CFTR gene.
Exclusivity End Date: 08/07/2025 
Exclusivity Protected Indication* :  ORKAMBI (lumacaftor/ivacaftor) oral granules indicated for the treatment of cystic fibrosis (CF) in patients age 2 through 5 years old who are homozygous for the F508del mutation in the CFTR gene.
4 Generic Name: lumacaftor and ivacaftor
Trade Name: Orkambi
Marketing Approval Date: 09/02/2022
Approved Labeled Indication: Treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene
Exclusivity End Date: 09/02/2029 
Exclusivity Protected Indication* :  Treatment of cystic fibrosis (CF) in patients aged 1 year to less than 2 years who are homozygous for the F508del mutation in the CFTR gene
*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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