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FDA Approves Symdeko to Treat Underlying Cause of CF in Children Ages 6-11 Years with Certain Mutations in CFTR Gene

The U.S. FDA approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6 through 11 years who have two copies of the F508del-CFTR mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator gene that is responsive to the Vertex Pharmaceuticals Inc. medicine.

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Vertex cystic fibrosis drug gets expanded approval for use in infants

U.S. health regulators expanded the use of Vertex Pharmaceuticals Inc.’s cystic fibrosis drug Kalydeco to include patients aged between 12 months and 24 months.

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FDA Approves Orkambi For New Cystic Fibrosis Use

The FDA approved Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) to include use in children ages 2-5 years with cystic fibrosis who have two copies of the F508del-CFTR mutation.

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Vertex Receives Expanded EU Approval for Orkambi

Vertex Pharmaceuticals Incorporated announced that the European Commission granted extension of the Marketing Authorization for Orkambi (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis in people with two copies of the F508del mutation, to include children ages 6 through 11.

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Corbus drug shows promise as add-on therapy for cystic fibrosis

An anti-inflammatory drug being developed by Corbus Pharmaceuticals Holdings as an add-on treatment for cystic fibrosis demonstrated promising safety and was well tolerated in a small midstage study, according to data released by the company.

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Vertex CF combo succeeds in late-stage trials

Vertex Pharmaceuticals Inc. said its Kalydeco cystic fibrosis treatment given with an experimental drug demonstrated significant improvements in lung function in a pair of late-stage trials the company plans to use to seek approval of the combination therapy.

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Horizon Pharma Buys Raptor in $800 Million Deal

Shares of Raptor Pharmaceutical Corp. were up after Horizon Pharma announced it had struck a deal to acquire the rare disease company for $800 million.

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Vertex Dumps Phase III Cystic Fibrosis Study

Vertex Pharmaceuticals Inc. announced it terminated clinical development of one late-stage trial studying the combination of its investigational cystic fibrosis compound VX-661 with the approved cystic fibrosis drug Kalydeco.

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June 2019 Focus: Payer access, biotech/biopharma, DTC, rare diseases, and more!


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