The Government has been urged to ensure that a breakthrough treatment for people living with the extremely painful skin disease, Epidermolysis bullosa (EB), is supplied to everyone who needs it, once it becomes available in Ireland
EB, also known as Butterfly Skin, is a rare, genetic condition which causes the skin to blister at the slightest touch.
Patients need their wounds regularly dressed with bandages – either every 24 or 48 hours – to prevent them from becoming infected. In severe cases, bandage changes can take up to 3 to 4 hours.
US researchers, Krystal Biotech, however, have discovered that a newly developed gene therapy gel helps to heal the blisters – with more than two-thirds (67%) of patients who took part in clinical trials reporting that their wounds healed completely after six months.
The team at Stanford University, California, is now seeking approval for the new drug from the Food and Drug Administration (FDA) in the United States.
Around 300 people in Ireland suffer with EB, and Jimmy Fearon, CEO of EB charity DEBRA Ireland, has called on the Government to ensure that the life-changing treatment is made available to everyone who needs it.
Mr Fearon said the FDA is expected to approve the new gel – called beremagene geperpavec (B-VEC) – in May.
It is hoped the approval process for the European market, via the European Union’s European Medicines Agency (EMA), will begin shortly after.
“This is a fantastic breakthrough and fantastic news for everyone in Ireland diagnosed with the condition, and their families,” he said.
“The whole process around the changing of bandages for people with EB is massively distressing for patients and carers alike, therefore any treatment which helps to alleviate the awful pain that EB sufferers endure is more than welcome.”
Participants in the B-VEC trial are reporting that the gel is a life-changer and large wounds they have had for years have either gone, or have greatly improved.
“I only hope that when the drug receives approval for use in this country, the Government does everything in their power to make sure it is made available and accessible to everyone in Ireland who needs it,” Mr. Fearon said.
Mr Fearon, however, stressed that the new drug was one step further in helping improve the quality of life of people living with EB – and that DEBRA Ireland remained committed to funding research into helping find an eventual cure.
“We hope the public continues to show their support for our efforts by donating via our website, debraireland.org,” he said.
People diagnosed with EB have a genetic mutation and are unable to produce the collagen VII protein, which binds the middle and outer layer of the skin together.
The blisters and wounds are caused by the layers ‘sliding’ across each other.
Suffers are vulnerable to infections and skin cancer and often die in early adulthood.
The new gel applies a copy of the collagen VII gene to strengthen the skin and prevent blisters forming on the surface.
Results from the latest B-VEC trials were published in the latest edition of the New England Journal of Medicine.