Patient advocates have welcomed the approval of a first treatment for Epidermolysis Bullosa (EB), also known as butterfly skin disease, by European medicines regulators.
Debra Ireland represents about 300 patients, many of them children, who suffer from the condition, which sees their skin blister at the slightest contact.
“It’s a groundbreaking achievement,” said Debra Ireland chief executive Jimmy Fearon. “Twelve years ago, there was nothing on the horizon and we were trying to encourage small bits of research at university level. We are overjoyed at the news that there will now be a treatment for EB.”
However, it remains unclear when patients in Ireland will get access to the drug, which has been developed by Irish rare drugs group Amryt Pharma. The drug, Filsuvez, must now undergo a health technology assessment — a value for money exercise — before a decision is taken on whether it will be made available to patients here.
“We have no idea what it will cost but you can be sure it will be expensive,” Mr Fearon said. He is hoping that, given the very small patient population, the cost involved will be seen as modest in relation to the medicines budget alongside the fact that there is no other treatment available.
But he acknowledges it may take some time. “This is a first treatment. We’ve never been here before. But when drugs are first approved, it can take some time before Irish patients get them,” he said.
“EB can affect every organ in the body except the brain,” Mr Fearon explained. “The worst part is the bandage changes. It is like having third-degree burns and it takes three hours a day.”
Amryt’s drug is not a cure but it has been shown in trials to accelerate the healing process for the skin when it does blister.
Meanwhile, Amryt said it will submit a formal dispute resolution request for the drug with the US regulator, the FDA. This is a path drug companies can use to try to seek to resolve scientific and/or medical disputes about new drug applications that cannot be resolved otherwise.