Chiesi presents birch triterpenes real-world EB data at PeDRA 2026
Chiesi Global Rare Diseases has presented three scientific and real-world evidence papers on birch triterpenes topical gel at the 14th Annual Conference of the Pediatric Dermatology Research Alliance (PeDRA), held 15–17 October 2026 in Alexandria, Virginia. The presentations cover new mechanistic data and clinical experience in patients with junctional and dystrophic epidermolysis bullosa (EB).
Birch triterpenes topical gel, marketed as Filsuvez, holds approval across three major jurisdictions: the European Commission granted approval in June 2022, the US FDA followed in December 2023, and the UK MHRA approved the product in September 2022 for the treatment of partial-thickness wounds in patients aged six months and older with junctional or dystrophic EB.
What the data show
Two of the three PeDRA presentations build on findings shared earlier in 2026. The first expands on mechanism-of-action data originally presented at the European Society for Dermatological Research annual meeting in September; the second adds to real-world experience of birch triterpenes used in combination treatment regimens, first discussed at the Society for Pediatric Dermatology meeting in July. A third presentation, new to the PeDRA platform, reports real-world observations on use of the gel in adolescents with chronic, lifelong wounds.
Lara Wine Lee, Pediatric Dermatologist at the Medical University of South Carolina, said that for EB, "real-world evidence can provide important insights into how wound management approaches are being used across different care settings," and that pairing clinical observations with mechanistic research helps identify questions warranting further study.
Rachele Berria, Senior Vice President of Medical Affairs at Chiesi Global Rare Diseases, framed the body of work as connecting laboratory science with clinical practice, noting that what is learnt about the compound's mechanism alongside clinician experience helps determine where research should go next.
Market and regulatory context
EB is a rare genetic disorder affecting an estimated 500,000 people globally, characterised by extreme skin fragility and wound burden that in severe forms carries risks of squamous cell carcinoma, sepsis and reduced life expectancy. It represents a small but clinically significant rare-disease niche where evidence generation remains challenging given patient numbers and disease heterogeneity.
The progressive build-up of real-world evidence across multiple conference platforms is a strategy increasingly adopted by rare-disease companies seeking to reinforce and extend clinical utility beyond the original registrational dataset. For Filsuvez, which was approved on a wound-healing endpoint rather than a broader disease-modification claim, the accumulation of combination-use and adolescent population data may carry commercial weight in markets where formulary access decisions rest partly on post-approval evidence packages.
Chiesi has also been expanding its rare-disease footprint more broadly. In 2026 the group acquired KalVista Pharmaceuticals, which develops oral therapies for rare diseases, signalling continued investment in the space beyond dermatology. That appetite for rare-disease assets places the EB franchise within a growing internal portfolio rather than as a standalone bet.
Alongside the scientific programme, Chiesi is marking EB Awareness Week (25–31 October) through its LivingWithEB digital platform and support for DEBRA International's Inside Out campaign. The company did not report new efficacy or safety data from controlled trials in this announcement.