On July 2, 2015 the FDA announced its approval for the potent duo of Lumacaftor and Ivacaftor to treat patients suffering from Cystic Fibrosis (CF). Coined Orkambi, which is marketed by Vertex Pharmaceuticals, this effectual treatment is specifically approved for patients ages 12 and older who have the homozygous F508 mutation of the CF transmembrane[…]
CF Therapy Combination Lumacaftor and Ivacaftor Approved
Drug Research & Development API, FDA Approved 2015, Respiratory Tract Agent Tags: CFCFTRcystic fibrosis Jul 24, 2015
Ivacaftor Receives MAA Line Extension from the EMA for Children with CF
Drug Research & Development API Tags: CFCFTRcystic fibrosis Jul 15, 2015
Known as the brand name Cystic Fibrosis drug Kalydeco, marketed by Vertex Pharmaceuticals, Ivacaftor recently received an MAA line extension. As of June 2015 children ages two through five who have specific mutations in the CFTR gene may be administered Ivacaftor as therapy. As a unique, tolerable and effectual treatment Ivacaftor acts as a novel[…]
Ivacaftor and Lumacaftor Create Positive Results for Common CF Mutation
Drug Research & Development API, NDA, Respiratory Tract Agent Tags: AustraliaCFcystic fibrosis Aug 07, 2014
Recently revealed results from a successful Phase 3 study combining Ivacaftor {Kalydeco} and Lumacaftor {VX-809} are extremely positive for patients with a common Cystic Fibrosis {CF} mutation. Patients who have the mutation of Cystic Fibrosis, which entails two copies of the F508del, have demonstrated in Phase 3 clinical trials an effective response to treatment with[…]
Ataluren Phase 3 Data Encouraging for Cystic Fibrosis
Drug Research & Development API Tags: AtalurenCFCFTRcystic fibrosis May 22, 2014
Ataluren, formerly known as PTC124 appears to be a viable treatment for Cystic Fibrosis. This oral compound targets CFTR, the protein product and faulty gene believed to be the cause of Cystic Fibrosis. Ataluren also appears to be particularly effective for patients with Cystic Fibrosis (CF) nonsense mutation. This genetic mutation interrupts the CFTR protein[…]
