Elexacaftor-Tezacaftor-Ivacaftor and Ivacaftor (Trikafta)
نویسندگان
چکیده

 CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well patient clinician perspectives, a drug or class.
 The inform non-binding recommendations that help guide decisions Canada's federal, provincial, territorial governments, with exception Quebec.
 This review assesses ELX-TEZ-IVA (Trikafta) ELX 100 mg, TEZ 50 IVA 75 mg (combination tablet), 150 (tablet); oral.
 Indication: Treatment cystic fibrosis in patients aged ? 12 years who have at least 1 F508del mutation transmembrane conductance regulator gene.
منابع مشابه
Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR.
RATIONALE Tezacaftor (formerly VX-661) is an investigational small molecule that improves processing and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) in vitro, and improves CFTR function alone and in combination with ivacaftor. OBJECTIVES To evaluate the safety and efficacy of tezacaftor monotherapy and of tezacaftor/ivacaftor combination therapy in subjects w...
متن کاملLumacaftor-ivacaftor (Orkambi) for cystic fibrosis: behind the 'breakthrough'.
Lumacaftor-ivacaftor (Orkambi) was recently approved by the Food and Drug Administration (FDA) in the USA to treat patients at least 12 years old who have cystic fibrosis due to two copies of the F508del (Phe508del) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Lumacaftor-ivacaftor has been designated a ‘breakthrough’ and is slated to cost $259 000 per year pe...
متن کاملIvacaftor potentiation of multiple CFTR channels with gating mutations.
BACKGROUND The investigational CFTR potentiator ivacaftor (VX-770) increased CFTR channel activity and improved lung function in subjects with CF who have the G551D CFTR gating mutation. The aim of this in vitro study was to determine whether ivacaftor potentiates mutant CFTR with gating defects caused by other CFTR gating mutations. METHODS The effects of ivacaftor on CFTR channel open proba...
متن کاملIvacaftor Therapy in CF Patients: Single Center Experience
Ivacaftor is the first novel cystic fibrosis pharmaceutical that acts at the molecular level to potentiate cystic fibrosis transmembrane conductance regulator (CFTR) function and was first approved for clinical use in 2012. We are sharing our single center experience of five patients: four from pediatric age group and one adult patient. All patients had both subjective and objective improvement...
متن کاملGrowth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor.
BACKGROUND AND OBJECTIVES Cystic fibrosis (CF) is known for its impact on the lung and pancreas of individuals; however, impaired growth is also a common complication. We hypothesized that targeting the biological defect in the CF transmembrane conductance regulator (CFTR) protein may affect growth outcomes. METHODS In this post hoc analysis, we assessed linear growth and weight in 83 childre...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: Canadian journal of health technologies
سال: 2021
ISSN: ['2563-6596']
DOI: https://doi.org/10.51731/cjht.2021.189