Patients with a distressing genetic condition that causes their skin to blister at the slightest touch have new hope after an Irish company secured European approval for a new drug.
European regulators have approved what is the first treatment for the thousands of patients – many of them very young children – who suffer from epidermolysis bullosa (EB), a condition sometimes known as butterfly skin.
Around 300 Irish children are among those who will benefit following the approval for the drug, Filsuvez for use in patients over the age of six months.
Amryt Pharma, the Irish company behind the new drug, specialises in developing therapies for rare diseases.
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Chief executive Joe Wiley said the European Commission approval of the therapy was “a major positive development for European patients who suffer from this debilitating condition”.
He said his company had the team and the financial flexibility to bring the drug to market swiftly. But it was unclear on Thursday just how long that might take and how much patients or the health service will have to pay for the treatment.
There are understood to be around 500,000 people suffering from EB worldwide, including 5,000 in the UK. Amryt estimates that the global market for EB is in excess of $1 billion.
The genetic condition affects around one in every 20,000 children. It causes a breakdown in the natural proteins that hold the skin together. Patients with severe forms of EB suffer from severe, chronic blistering, ulceration and scarring of the skin. They are also more prone to a form of skin cancer, infections and the risk of premature death.
Shares in Amryt jumped over 7 per cent on the news.
“EB is a rare and distressing genetic skin disorder affecting young children and adults for which, until now, there has been no approved treatment in any market,” Amryt said.
The European Commission approval will allow the company sell the drug across the EU as well as in Iceland, Liechtenstein and Norway. The company said the Europe green light also gives the company documentation that could be the basis for future applications for approval in Latin America and the Middle East.
The European approval for Filsuvez came on the back of the largest ever global trial conducted among EB patients at 58 sites in 28 countries.
Dr Wiley has previously referred to impending European approval as “the most significant milestone in Amryt’s history” and “a major positive development for European patients that suffer from this debilitating condition”.
“This is the culmination of years of hard work from all the Amryt team. I’d like to formally thank both them and all the patients, care givers and physicians for their commitment and efforts in getting us to this point,” he said.
