EADV Congress 2023: A new era for epidermolysis bullosa

A new era for epidermolysis bullosa

Four experts discuss investigational treatments for epidermolysis bullosa (EB), updates in the field and their experience from clinical practice in interviews at EADV and pre-recorded presentations, chaired by Professor Cristina Has.

Urgent gaps in the knowledge of EB – Closing the gaps – EADV 2023 data: Impact for people living with EB

EB is a group of genetic disorders, characterised by blistering of skin, with a devastating impact on quality of life^1,2. Professor Has (University of Freiburg, Germany) tells us about some of the most urgent gaps in the field of EB that still need addressing, the information needed to close these gaps and her highlights from EADV 2023.

European Commission and US Food and Drug Administration (FDA) approval of birch bark extract (EC) / birch triterpenes (FDA) for partial thickness wounds associated with dystrophic EB (DEB) and junctional EB (JEB), and FDA approval of beremagene geperpavec (B-VEC) for DEB in people aged 6 months or older pave the way for a new era of EB treatment^3-5 

Now that EB is on the map, how can we continue to share knowledge, and what can we expect from future developments?

Developments in EB in the next 2 years – Hybridisation of expertise in EB –The importance of educational initiatives in EB – A new era for EB

Professor Has introduces the pre-recorded presentations of the session at EADV 2023 and gives an overview of EB as a disease.

Introduction to session – Introduction to epidermolysis bullosa

References

1. Has C, Bauer JW, Bodemer C, Bolling MC, Bruckner‐Tuderman L, Diem A, et al. Consensus reclassification of inherited epidermolysis bullosa and other disorders with skin fragility. British Journal of Dermatology. 2020;183(4):614-627.
2. Bardhan A, Bruckner-Tuderman L, Chapple ILC, Fine JD, Harper N, Has C, et al. Epidermolysis bullosa. Nat Rev Dis Primers. 2020;6(1):78.
3. Filsuvez (birch bark extract) summary of product characteristics. 2022. Available at: https://www.ema.europa.eu/en/documents/product-information/filsuvez-epar-product-information_en.pdf.
4. U.S. Food & Drug Administration. FDA approves first topical gene therapy for treatment of wounds in patients with dystrophic epidermolysis bullosa. https://www.fda.gov/news-events/press-announcements/fda-approves-first-topical-gene-therapy-treatment-wounds-patients-dystrophic-epidermolysis-bullosa. Accessed 18 October.
5. Filsuvez (birch bark extract) prescribing information. 2023. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215064s000lbl.pdf.
6. Gretzmeier C, Pin D, Kern JS, Chen M, Woodley DT, Bruckner-Tuderman L, et al. Systemic collagen VII replacement therapy for advanced recessive dystrophic epidermolysis bullosa. Journal of Investigative Dermatology. 2022;142(4):1094-1102.e1093.
7. Nyström A, Bornert O, Kühl T, Gretzmeier C, Thriene K, Dengjel J, et al. Impaired lymphoid extracellular matrix impedes antibacterial immunity in epidermolysis bullosa. Proc Natl Acad Sci U S A. 2018;115(4):E705-e714.
8. Dieter K, Niebergall-Roth E, Daniele C, Fluhr S, Frank NY, Ganss C, et al. ABCB5+ mesenchymal stromal cells facilitate complete and durable wound closure in recessive dystrophic epidermolysis bullosa. Cytotherapy. 2023;25(7):782-788.
9. Kiritsi D, Dieter K, Niebergall-Roth E, Fluhr S, Daniele C, Esterlechner J, et al. Clinical trial of ABCB5+ mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight. 2021;6(22).
10. NHS. Great Ormond Street Hospital for Children. Clinical trial for children with rare skin condition gets underway. https://www.gosh.nhs.uk/news/clinical-trial-for-children-with-rare-skin-condition-gets-underway/. Accessed 18 October.
11. Kern JS, Sprecher E, Fernandez MF, Schauer F, Bodemer C, Cunningham T, et al. Efficacy and safety of Oleogel-S10 (birch triterpenes) for epidermolysis bullosa: results from the phase III randomized double-blind phase of the EASE study. British Journal of Dermatology. 2022;188(1):12-21.
12. Murrell D, L. Bruckner A, Sprecher E, Kiritsi D, Maher L, Cunningham T, Kern JS. BG04 Oleogel-S10 (birch triterpenes) in the treatment of epidermolysis bullosa wounds: 24-month efficacy and safety data from the EASE study. British Journal of Dermatology. 2023;188(Supplement_4).
13. Torres-Pradilla M, Álvarez E, Novoa M, Lozano I, Trujillo M. LB1791 Oleogel-S10 (birch bark extract) in dystrophic epidermolysis bullosa: A case series evaluating the impact on wound burden over 2 years. Journal of Investigative Dermatology. 2023;143(9):B34.