Two new medicines could transform the treatment of epidermolysis bullosa (EB) – often called “butterfly skin” – in Ireland. Debra, the charity which works to support those with the skin disorder, where the skin is extremely fragile and blisters easily, says the entry of Filsuvez into the Irish market will ease the extreme pain endured by those with ‘Butterfly Skin’.
The condition occurs when there is an absence of the proteins needed to bind different layers of the skin together. This creates excruciating blistering and tearing with the slightest touch or movement. The most serious cases of EB require bandaging every 24 hours to prevent infection.
The disease is rare, affecting around 300 people in Ireland, and Debra says it is often overlooked when it comes to medical research, which has resulted in a need for research to be done. Debra is currently funding EB research projects in Ireland in the hope that it can move the needle forward for a potential new combination of treatments for the chronic condition.
The expected introduction of the prescription medical gel before the end of the year could pave the way for gene therapy treatments, such as Vyjuvek, Debra said.
In 2023, the US Food and Drug Administration granted approval of VYJUVEK is based on two clinical studies. VYJUVEK became available in the US the following year.
“Where Filsuvez is a promising start, as it can offer temporary pain relief, we hope it will mark just the beginning for the treatment of rare disease in Ireland,” – Sinead Hickey, Head of Research at Debra, said on Monday.
“Vyjuvek has the potential to provide life-changing wound healing for people living with Dystrophic EB – it has already proven to be very effective in the US,” the organidation said.
A topical gel, it delivers new COL7A1 genes directly to skin wounds to promote wound healing.
“A huge benefit is that it does not have to be applied by a medical practitioner but can be done – using only a credit card sized amount – by a trained individual or family member.”
“Unfortunately, Ireland is one of the slowest countries in Europe to move from recommendation to market and it could still be between seven and 12 years before we see Vyjuvek available to people here.”
Filsuvez, meanwhile, is applied to a sterile non-adhesive wound dressing or directly to the skin to ease the extreme pain of wounds caused by EB.
Debra made submissions to the NCPE (National Centre for Pharmacoeconomics), which has recommended the HSE provide the gel once agreement is reached on pricing. A timeline has yet to be announced but Debra says it is optimistic a resolution will be found by the end of summer.
“The treatment for EB hasn’t advanced much over time, so new treatments are always bring hope,” CEO of Debra, Jimmy Fearon, said. Mr Fearon ssaid that while the treatments are not a cure for the condition, it may reduce the equivalent of a bandage change every two weeks, providing some pain alleviation.
“What this primarily represents is hope – breaking through the glass ceiling would hopefully mean that others will follow,” he said, adding:
“Debra and other EB charities around the world support and invest in important research but, ultimately, it’s going to be the pharmaceutical companies and venture capitalists that run the breakthrough.”
“What we really need is faster progression from recommendation to market – time is of the essence for the 300 people in Ireland living in pain due to this condition.”
Debra has reiterated calls for government help – saying that for the cost of two coffees per person living with EB in Ireland per day, a real difference can be made in the lives of those with the genetic condition.
Last year, patient ambassador for Debra Ireland, Emma Fogarty, took on the Dublin Marathon with actor Colin Farrell, who she met 15 years ago through the charity. Fogarty, 40, is the oldest surviving person with the condition in Ireland. While Debra had expressed disappointment that the Government had “failed to prioritise” EB care for the second year in a row in the 2024 budget, Mr Farrell seemed to capture the attention of the Government – meeting with then-Taoiseach Simon Harris after he completed the marathon.