CF Drugs Update: January 2019

CF Drugs Update: January 2019

Orkambi, Kalydeco and Smykevi: More Good News

ORKAMBI

Cystic Fibrosis Ireland welcomes the news that Orkambi has been approved by the EMA (European Medicines Agency) for children aged 2-5 years old with 2 copies of the F508del gene alteration. 

Background:
Orkambi was originally approved for reimbursement in Ireland for 12 years and older in 2017. Orkambi was subsequently extended to 6-11 year olds in 2018. The further extension of Orkambi to 2-5 years olds approved by the EMA is consistent with the drugs portfolio deal agreed in April 2017 between the HSE and Vertex Pharmaceuticals. 

CFI will seek clarification from the HSE on timing of availability of the latest Orkambi extension in Ireland. 


SYMKEVI

CF Ireland understands that the introduction of Symkevi (approved on 1 January 2019) is going very well. A small number of patients have been switched to Symkevi who were originally on Orkambi, based on clinical criteria and expert advice from Consultants. However CFI also understands that the vast majority of those on Orkambi will remain on Orkambi as CFI predicted in earlier news updates.

Background:
Symkevi is a CFTR medication that is a possible alternate for those eligible for Orkambi and also for a small number of other linked gene alterations


KALYDECO

The EMA have also recently approved the extension of Kalydeco to 1-2 year olds. 

Cystic Fibrosis Ireland is also seeking clarification if Kalydeco will be extended to those under 18 years of age with the R117H gene alteration. 

Background:
Kalydeco was the first CFTR drug introduced to Ireland in 2013 of which Orkambi and Symkevi have since followed (2017 and 2019). These are the first drugs that treat the underlying cause of CF. They are personalised drugs in the sense that they are tailored for those with certain gene alterations.

Kalydeco impacts on those with the G551D gene alteration (15% of the CF population in Ireland) but also other named gene alterations including R117H (6% of the CF population in Ireland). The most common gene alteration in Ireland and worldwide is people with CF with 2 copies of the F508del gene alteration (about 57% in Ireland). This is the group of patients eligible for Orkambi and Symkevi