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Vertex’s Orkambi (lumacaftor/ivacaftor) Receives EMA’s CHMP Positive Opinion for Children With Cystic Fibrosis Ages 1 to <2 Years

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Vertex’s Orkambi (lumacaftor/ivacaftor) Receives EMA’s CHMP Positive Opinion for Children With Cystic Fibrosis Ages 1 to <2 Years

Shots:

  • The EMA’s CHMP has adopted a positive opinion for the label extension of Orkambi (lumacaftor/ivacaftor) to treat children with CF aged 1-<2yrs. who have two copies of the F508del mutation in the CFTR gene
  • The expanded approval was based on the P-III clinical trial evaluating Orkambi in children aged 1-<2yrs. which was found to be safe and well tolerated, and reduced the sweat chloride concentration
  • Orkambi was approved in the EU for the treatment of patients with CF aged ≥2yrs. who have two copies of the F508del mutations. If Orkambi is approved, a treatment option will be available for the first time for about 300 young children with CF

Ref: Businesswire Image: Vertex

Related News:- Vertex’s Orkambi (lumacaftor/ivacaftor) Receives Health Canada’s Market Authorization for Children with Cystic Fibrosis Aged 1 to <2 Years

 

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Senior Editor

Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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