Suggested remit - To appraise the clinical and cost effectiveness of lumacaftor in combination with ivacaftor within its marketing authorisation for treating cystic fibrosis in children ages 2 to 11 years old who are homozygous for the F508del mutation
Following on from information provided to NICE by the company in October 2019, the appraisal of Lumacaftor with ivacaftor for treating cystic fibrosis in children aged 2 to 11 homozygous for the F508del mutation [ID1486] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued.
 
Status Discontinued
Technology type Medicine
Decision Selected
Process TA
ID number 1486

Project Team

Project lead Kate Moore

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Timeline

Key events during the development of the guidance:

Date Update
25 January 2023 Discontinued. Following on from information provided to NICE by the company in October 2019, the appraisal of Lumacaftor with ivacaftor for treating cystic fibrosis in children aged 2 to 11 homozygous for the F508del mutation [ID1486] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued.
07 October 2019 Suspended. This appraisal was due to start at the end of September. However the company, Vertex Pharmaceuticals, has informed us that it will not be participating in the appraisal. As a consequence, we have now suspended this appraisal. Please monitor this webpage for further updates.
22 August 2019 Draft scope documents
25 September 2018 - 23 October 2018 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

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