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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
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
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:  lumacaftor/ivacaftor
Trade Name:  Orkambi
Date Designated:  06/30/2014
Orphan Designation:  Treatment of cystic fibrosis
Orphan Designation Status:  Designated/Approved
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.
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:  lumacaftor/ivacaftor
Trade Name:  Orkambi
Date Designated:  06/30/2014
Orphan Designation:  Treatment of cystic fibrosis
Orphan Designation Status:  Designated/Approved
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
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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