Cystic Fibrosis Archives

Vertex prices cystic fibrosis combo treatment at $311,503 per year

Approvals, Blockbusters, Business, Cystic Fibrosis, England, FDA, FDA/Regulatory, Genetic Diseases, Genetic Mutations, New Drug Application (NDA), Proteins, Sales Guidance, Shares, United States

Vertex Pharmaceuticals Inc. priced the company’s FDA-approved Trikafta, a three-drug combination for cystic fibrosis, at $311,503 per year.

October 21, 2019/by Reuters Health

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

CFTR protein, Children, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), FDA, FDA/Regulatory, Genetic Diseases, New Indications

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.

June 21, 2019/by Business Wire

FDA Approves Orkambi For New Cystic Fibrosis Use

Approvals, Children, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), FDA/Regulatory, First-of-its kind, Genetic Diseases, New Indications

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.

August 7, 2018/by Business Wire

Vertex Receives Expanded EU Approval for Orkambi

Approvals, BMI, Cystic Fibrosis, European Commission, Expanded Approval, Genetic Diseases

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.

January 10, 2018/by Business Wire