Latest news with #Vyjuvek
Belfast Telegraph
18-05-2025
- Health
- Belfast Telegraph
Cutting-edge medicines hoped to transform treatment of incurable skin condition
Debra, which supports 300 people living with epidermolysis bullosa (EB) in Ireland, said the entry of Filsuvez into the Irish market will ease the extreme pain endured by those with 'Butterfly Skin'. The disease is caused by the absence of proteins between the skin layers, making the skin ultra fragile and easy to wound. The charity insists that 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. Sinead Hickey, head of research at Debra, said: 'Where Filsuvez is a promising start, as it can offer temporary pain relief, we hope it will mark the beginning for the treatment of rare disease in Ireland. '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 topical gel delivers new COL7A1 genes directly to skin wounds to promote wound healing. Ms Hickey said it does not have to be applied by a medical practitioner but can be done 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,' she said. 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 provides the gel once agreement is reached on pricing. A timeline has yet to be announced but Debra is optimistic a resolution will be found by the end of summer. The charity's chief executive, Jimmy Fearon, said its entry into the Irish market is a step in the right direction. 'The treatment for EB hasn't advanced much over time, so new treatments always bring hope,' he said. 'This is not a cure but 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. 'Debra and other EB charities around the world support and invest in important research but, ultimately, it's the pharmaceutical companies and venture capitalists that run the breakthrough.' Ireland is in the lowest quartile in Europe for introducing new drugs to the market, the chief executive said. 'It's an arduous process to bring a drug to market and this is a positive step,' added Mr Fearon. '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.'
The Journal
18-05-2025
- Health
- The Journal
Cutting-edge medicines hoped to transform treatment of incurable skin condition EB
THE INTRODUCTION OF two new cutting-edge medicines are hoped to transform the treatment of a rare incurable skin condition, a charity has said. Debra, which supports 300 people living with epidermolysis bullosa (EB) in Ireland, said the entry of Filsuvez into the Irish market will ease the extreme pain endured by those with 'Butterfly Skin'. The disease is caused by the absence of proteins between the skin layers, making the skin ultra fragile and easy to wound. The charity insists that 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. Sinead Hickey, head of research at Debra, said: 'Where Filsuvez is a promising start, as it can offer temporary pain relief, we hope it will mark the beginning for the treatment of rare disease in Ireland. '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.' Actor Colin Farrell with his friend Emma Fogarty, running in the Irish Life Dublin Marathon in Dublin in October, to raise money for people living with Epidermolysis Bullosa (EB), a rare genetic skin condition suffered by Ms Fogarty. Alamy Stock Photo Alamy Stock Photo The topical gel delivers new COL7A1 genes directly to skin wounds to promote wound healing. Ms Hickey said it does not have to be applied by a medical practitioner but can be done 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,' she said. Advertisement 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 provides the gel once agreement is reached on pricing. A timeline has yet to be announced but Debra is optimistic a resolution will be found by the end of summer. The charity's chief executive, Jimmy Fearon, said its entry into the Irish market is a step in the right direction. 'The treatment for EB hasn't advanced much over time, so new treatments always bring hope,' he said. 'This is not a cure but 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. 'Debra and other EB charities around the world support and invest in important research but, ultimately, it's the pharmaceutical companies and venture capitalists that run the breakthrough.' Ireland is in the lowest quartile in Europe for introducing new drugs to the market, the chief executive said. 'It's an arduous process to bring a drug to market and this is a positive step,' added Mr Fearon. '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.'
Irish Daily Mirror
18-05-2025
- Health
- Irish Daily Mirror
Entry of new medicine into Irish market to ease pain of incurable skin condition
The introduction of two new cutting-edge medicines are hoped to transform the treatment of a rare incurable skin condition, a charity has said. Debra, which supports 300 people living with epidermolysis bullosa (EB) in Ireland, said the entry of Filsuvez into the Irish market will ease the extreme pain endured by those with 'Butterfly Skin'. The disease is caused by the absence of proteins between the skin layers, making the skin ultra fragile and easy to wound. The charity insists that 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. Sinead Hickey, head of research at Debra, said: "Where Filsuvez is a promising start, as it can offer temporary pain relief, we hope it will mark the beginning for the treatment of rare disease in Ireland. "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 topical gel delivers new COL7A1 genes directly to skin wounds to promote wound healing. Ms Hickey said it does not have to be applied by a medical practitioner but can be done 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," she said. 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 provides the gel once agreement is reached on pricing. A timeline has yet to be announced but Debra is optimistic a resolution will be found by the end of summer. The charity's chief executive, Jimmy Fearon, said its entry into the Irish market is a step in the right direction. "The treatment for EB hasn't advanced much over time, so new treatments always bring hope," he said. "This is not a cure but 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. "Debra and other EB charities around the world support and invest in important research but, ultimately, it's the pharmaceutical companies and venture capitalists that run the breakthrough." Ireland is in the lowest quartile in Europe for introducing new drugs to the market, the chief executive said. "It's an arduous process to bring a drug to market and this is a positive step," added Mr Fearon. "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."
BreakingNews.ie
18-05-2025
- Health
- BreakingNews.ie
Cutting-edge medicines hoped to transform treatment of incurable skin condition
The introduction of two new cutting-edge medicines are hoped to transform the treatment of a rare incurable skin condition, a charity has said. Debra, which supports 300 people living with epidermolysis bullosa (EB) in Ireland, said the entry of Filsuvez into the Irish market will ease the extreme pain endured by those with 'Butterfly Skin'. Advertisement The disease is caused by the absence of proteins between the skin layers, making the skin ultra fragile and easy to wound. The charity insists that 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. Sinead Hickey, head of research at Debra, said: 'Where Filsuvez is a promising start, as it can offer temporary pain relief, we hope it will mark the beginning for the treatment of rare disease in Ireland. '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.' Advertisement Sinead Hickey, head of research at Debra, says Filsuvez is a promising start. Photo: Alejandra Livschitz/Debra The topical gel delivers new COL7A1 genes directly to skin wounds to promote wound healing. Ms Hickey said it does not have to be applied by a medical practitioner but can be done 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,' she said. 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. Advertisement Debra made submissions to the NCPE (National Centre for Pharmacoeconomics), which has recommended the HSE provides the gel once agreement is reached on pricing. A timeline has yet to be announced but Debra is optimistic a resolution will be found by the end of summer. The charity's chief executive, Jimmy Fearon, said its entry into the Irish market is a step in the right direction. 'The treatment for EB hasn't advanced much over time, so new treatments always bring hope,' he said. Advertisement 'This is not a cure but 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. 'Debra and other EB charities around the world support and invest in important research but, ultimately, it's the pharmaceutical companies and venture capitalists that run the breakthrough.' Ireland Gel therapy to treat 'butterfly skin' condition we... Read More Ireland is in the lowest quartile in Europe for introducing new drugs to the market, the chief executive said. Advertisement 'It's an arduous process to bring a drug to market and this is a positive step,' added Mr Fearon. '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.'
Yahoo
30-04-2025
- Business
- Yahoo
FDA approves Abeona's $3.1m cell therapy for rare skin disease
Abeona Therapeutics has secured US Food and Drug Administration (FDA) approval for Zevaskyn (prademagene zamikeracel), a gene-corrected cell therapy designed to treat recessive dystrophic epidermolysis bullosa (RDEB). The approval follows years of clinical development and regulatory setbacks, and positions Abeona as the second company to bring a therapy for this condition to the US market. Also known as pz-cel, Zevaskyn is indicated for both paediatric and adult patients with RDEB. The therapy is administered as credit card-sized sheets of skin made from the patient's own keratinocytes, which are harvested, genetically modified to express the functional COL7A1 gene, and surgically applied to chronic wounds. The COL7A1 gene encodes a type of collagen essential for anchoring skin layers – something patients with RDEB lack due to genetic mutations. The FDA had previously rejected an application from Abeona last year, citing outstanding manufacturing and quality control (QC) concerns. Following a complete response (CR) letter and further chemistry, manufacturing and controls (CMC) submissions, the agency granted approval based on results from a pivotal Phase III study (NCT04227106). Despite a partial clinical hold in 2019, the study met both co-primary endpoints of wound healing and pain reduction, with no serious treatment-related adverse events (AEs) reported. The company also provided confirmatory evidence from a Phase I/IIa trial (NCT01263379), which supported the durability of healing after a single application. Zevaskyn is priced at $3.1m, placing it among the most expensive therapies in the US but in line with other rare disease gene and cell therapies. Abeona expects to make the product available commercially in Q3 2025. The company raised $35m in late 2022 after announcing the Phase III data, followed by a $25m financing round in July 2024 to support manufacturing and launch readiness. Krystal Biotech's Vyjuvek (beremagene geperpavec), a topical gene therapy gel for DEB, was approved in 2023 and generated $290.5m in revenue in 2024. Unlike Zevaskyn, which is applied once through surgery, Vyjuvek is dosed weekly and costs around $631,000 per patient per year. Vyjuvek also delivers a functional COL7A1 gene, though via a re-dosable, non-integrating viral vector. In the 29 April announcement, Abeona's CEO Vish Seshadri, said: 'We have heard from the RDEB community that there is a persistent unmet need to reliably address RDEB wounds, especially those that are chronic and prone to infection. Through a single surgical application, Zevaskyn can now offer people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds, as evidenced by the results from our pivotal Phase III study.' In connection with the approval, Abeona received a rare paediatric disease priority review voucher (PRV), which the company said it plans to monetise. The PRV programme, which grants expedited FDA review for a future drug application, is set to expire soon unless renewed by Congress. Rare disease advocates have warned that the programme's lapse, combined with existing FDA resource constraints, could deter future investment in paediatric rare disease research and development. "FDA approves Abeona's $3.1m cell therapy for rare skin disease" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
