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Vertex gains EU approval for earlier Orkambi use

Cystic Fibrosis drug approved in children aged 2-5 years


The European Commission has green lighted a label update for Vertex's cystic fibrosis (CF) drug Orkambi, which can now be used to treat children aged 2-5 years old.

The label boost is approved for those with the most common form of the disease, which is individuals who have inherited two copies of the defective F508del mutation. This makes Orkambi the first treatment available in Europe to treat these young children whose CF, who number around 1,500 in the EU.

Cystic Fibrosis is a life shortening disease, with the median age of death in the mid-to-late twenties. The hope is that earlier treatment will ultimately help extend lives, although the company has yet to generate this kind of long-term data.  The new trial in younger children focused on safety and efficacy as measured by changes in sweat chloride levels, However these are not related to changes in lung function, the kind of clinically important data which European HTA organisations will want to see.

Orkambi is a combination of lumacaftor and ivacaftor, which together targets the lack of CFTR proteins in CF patients.

“The approval by the European Commission brings us one step closer to our goal of bringing treatment to all people living with CF,” said Reshma Kewalramani, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex.


Reshma Kewalramani, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer, Vertex

‘‘By treating the underlying cause of disease early, we can potentially modify its course and offer patients the chance of improved outcomes.’’

The approval was based on phase 3 data, whereby Orkambi was general well tolerated for 24 weeks in 60 patients between the ages of 2-5. The safety profile was also consistent with that in patients aged 6 years and older.

Orkambi becomes the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis for this subset of patients, but not everyone will have access to it.

Vertex has spent the last two years tangled in a market access feud with NHS England over access to Orkambi. This culminated last year with MPs threatening to reveal all details of the negotiations.

While the MPs have backed down on this demand for the meantime, there is still no agreement in England, which has around 10,000 patients – the second highest population of CF patients in the world.

It’s a big market for Vertex, but it’s not budging on offering a further discount. Vertex is understood to have already offered NICE a discount of almost 90%, dropping the price of Orkambi from around £100,000 per patient to around £14,000 per patient.

Article by
Gemma Jones

22nd January 2019

From: Regulatory



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