Co Down mum of tot with Cystic Fibrosis ‘delighted’ with first European treatment drug Kalydeco – Belfast Live

A Co Down mum has welcomed positive movement on the first and only approved medicine in Europe that can treat the underlying cause of Cystic Fibrosis in some patients.

Jen Banks, whose tot Lorcn Maguire suffers from the genetic condition, says the news has brought precious hope at a time when CF patients are shielding from Coronavirus.

Vertex Pharmaceuticals said on Friday, Kalydeco (ivacaftor) was given a green light by the European Medicines Agencys Committee for Medicinal Products for Human Use for babies as young as six months.

Jen told Belfast Live: I think its wonderful!

The earlier people with CF begin to take modulators the better!

As CF is progressive these drugs will help to slow down the irreparable damage that it causes throughout the body.

The world is changing so much for everyone at the moment and for those with CF these life saving drugs have never been more important.

People with CF in the UK are having to follow shielding measures as they are regarded as being at extremely high risk of serious illness from Covid-19, anything that can help them should be welcomed with open arms.

It must be so wonderful for parents of children newly diagnosed with the R117H variation to hear that they need only wait six months before their babies can have Kalydeco.

The positive opinion on the medicine, which treats CF patients with the R117H mutation, will now be considered for approval by the European Commission.

If it gets the go ahead, Vertex says around 290 eligible patients across the UK will have access to Kalydeco within weeks of marketing authorisation as it will be reimbursed through the various agreements signed between them and UK health authorities in late 2019.

Carmen Bozic, MD, Executive Vice President and Chief Medical Officer at Vertex, said: This milestone also brings us one step closer to achieving our ultimate goal of bringing medicines forward to all people with CF.

Jen, whose three-year-old, takes Orkambi which she says has made a massive difference to Lorcns energy, appetite, weight gain, and digestion and he is a much happier wee man, even with all the treatment for the pseudomonas infection he smiles and laughs so much more and even sleeps better.

She is however eagerly waiting for Trikafta and added the 6-11 trials are currently running in spite of Covid-19, which is wonderful, and hopefully will be quickly followed by 2-5 trials.

There are still so many people with CF waiting for a drug that can help them, she continued.

In ROI there is a deal in place so that as soon as the EMA licenses the newest drug in the pipeline, Trikafta, everyone who is eligible (around 90% of people with CF) over the age of 12 will have access.

We have nothing like this in place for Trikafta and I would urge the NI Executive, the HSCB, and the drug company Vertex to work quickly to put a similar deal in place as soon as, if not before, Trikafta is approved by NICE.

In these uncertain times hope is the most precious thing weve got and should not be underestimated.

Its with delight that I think of the hope this news (about Kalydeco) will bring to families across NI.

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Co Down mum of tot with Cystic Fibrosis 'delighted' with first European treatment drug Kalydeco - Belfast Live