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

PMEA Awards 2020

COVID-19 Updates and Daily News

Featured jobs

PMHub

Add my company
Windrose Consulting Group

Founded in 2015, Windrose Consulting Group is a privately-held global strategy firm that partners with life science companies to maximize...

Latest intelligence

InSite Events™ National Pharma Meeting: A Customer Story
How our client leveraged Impetus Digital’s white-glove service, 360° coverage and care, and award-winning InSite Events™ platform to successfully transfer their annual national meeting online....
Fear or encouragement, which is best to drive behaviour change?
The communications industry depends on fear of the perceived loss of something, in order to create the hope found in a product benefit. But, if if left too long unchecked,...
The unheard, unseen, and uncorrected effects of COVID-19
The disproportionate effects of the pandemic have been highlighted amongst ethnic minorities, so is it likely that ethnic minorities will be more worried about participating? And could this exacerbate inequalities...

Infographics