Scottish Medicines Consortium does not back Vertex’ cystic fibrosis drugs Orkambi, Kalydeco

pharmafile | May 10, 2016 | News story | Manufacturing and Production, Research and Development Kalydeco, Orkambi, SMC, Vertex Pharma, cystic fibrosis, regulation 

The Scottish Medicines Consortium did not back two cystic fibrosis therapies from Vertex Pharmaceutical (Nasdaq: VRTX). 

SMC did not recommend Lumacaftor/Ivacaftor (Orkambi) and Ivacaftor (Kalydeco) due to the cost and uncertainty around long-term impact. 

Orkambi is not recommended for people over 12 and the use of Kalydeco will not be extended for 2-5 year olds, a drug which is currently already provided to people over six across Scotland. 

Simon Bedson, senior vice president at Vertex, said: “Vertex is disappointed that the SMC has not recommended Orkambi for people with cystic fibrosis ages 12 and older who have two copies of the F508del mutation or Kalydeco for children with CF ages 2 to 5 who have gating mutations for use within NHS Scotland.” 

In March, Nice issued draft guidance not recommending Orkambi for use on the National Health Service (NHS) in England. 

Nice said its independent appraisal committee had concluded that the cost of Orkambi was considerably higher than the current standard of care and it could not be considered a cost effective use of NHS resources. Nice noted that the drug costs £104,000 per year per patient and that this cost would be exacerbated by most patients remaining on currently-available treatments while taking Vertex’s drug.

SMC is an independent organisation that advises the NHS Health Boards about medicines in Scotland.  

Cystic fibrosis is a rare, life-threatening genetic disorder that causes progressive damage to a number of organs throughout the body. In Scotland, about half of people with the disease die by the age of 26. 

Anjali Shukla

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