Please login to the form below

Not currently logged in
Email:
Password:

Vertex’s Orkambi receives EU green light for licence extension

Becomes first medicine to treat the underlying cause of CF in children

VertexVertex has won a green light in Europe for a licence extension that makes it the first approved medicine to treat the underlying cause of CF in children.

The US biotech’s Orkambi is now the first medicine approved in the region to treat 6-11 year olds who carry two copies of the f580del mutation.

Orkambi, a twice-daily drug, is a combination of lumacaftor and ivacaftor, designed to enhance the function of the CFTR protein once it reaches the cell surface.

Patients with CF carry little-to-no CFTR protein at the cell surface due to inheriting two defective CFTR genes and according to Vertex, there are approximately 3,400 6-11 year olds who carry two copies of the mutation.

Simon Bedson, senior vice president and international general manager at Vertex was keen to highlight the market access work the company has undertaken to help it reach patients.

He said: “The innovative long-term agreements we have reached in countries like Ireland will enable eligible children to have rapid access to Orkambi.

“Where these agreements are not in place, Vertex is committed to working with local authorities so those who could benefit from this medicine are able to do so as quickly as possible.”

Orkambi’s new approval was based on data from two phase III studies showing a statistically significant improvement in lung function among children treated with the medication compared to placebo.

Article by
Gemma Jones

11th January 2018

From: Regulatory

Share

Tags

Featured jobs

Subscribe to our email news alerts

PMHub

Add my company
Kendle Healthcare

Company founder Neil Kendle was a pioneer in opinion leader engagement. In 2003, Neil brought together a small, dedicated team...

Latest intelligence

translational pharmacology medicine dose
The gold standard of scientific evidence
Will randomised controlled trials become a thing of the past?...
What does programmatic advertising mean for your pharma marketing strategy?
Senior Associate Consultant Jiayi Chen explains the benefits and pitfalls of programmatic advertising and reveals how it can impact return on investment in your marketing campaigns....
Discover who are the most engaging Pharma companies on Twitter
With engagement such a key performance metric we first created a weighted formula, incorporating likes, retweets and replies, which produced an engagement score for each account....

Infographics