Long‐Term Efficacy and Safety of Oleogel‐ S10 (Birch Triterpenes) for Pediatric Patients With Epidermolysis Bullosa

BACKGROUND/OBJECTIVES: Pediatric patients with epidermolysis bullosa (EB) experience lifelong complications, and wound healing is an important treatment goal. In the phase III EASE study (NCT03068780), Oleogel-S10 accelerated wound healing in EB. This prespecified subgroup analysis evaluated the long-term efficacy and safety of Oleogel-S10 in patients aged < 18 years from EASE. METHODS: EASE comprised a 90-day double-blind phase (DBP) and 24-month open-label phase (OLP). In the DBP, patients were randomized 1:1 to receive Oleogel-S10 or control gel; all patients could enter the OLP and receive Oleogel-S10. Endpoints in this analysis included wound healing, total body wound burden, dressing change burden, and safety. RESULTS: Overall, 156 pediatric patients were enrolled into the DBP (Oleogel-S10: n = 74; Control gel: n = 82); 147 entered the OLP. Oleogel-S10 resulted in 44.6% of pediatric patients with first complete target wound closure within 45 days, versus 25.6% with control gel (relative risk [95% CI]: 1.70 [1.11, 2.60], p = 0.012). Mean absolute body surface area percentage scores were 12.5% at DBP baseline and 5.3% at OLP Month 24 for the Oleogel-S10 group. Significantly more patients treated with Oleogel-S10 (38%) no longer required daily dressing changes versus control gel (9%) at DBP Day 90. Similar proportions of patients experienced adverse events in the Oleogel-S10 and control gel groups in the DBP, with most mild or moderate. CONCLUSIONS: These analyses of pediatric patients demonstrated accelerated wound healing, reduced frequency of dressing changes, and sustained reduction in wound burden up to 27 months. Long-term safety was consistent with the overall EASE population. TRIAL REGISTRATION: NCT03068780, EudraCT 2016-002066-32; registered 3 March 2017; https://clinicaltrials.gov/study/NCT03068780 and https://www.clinicaltrialsregister.eu/ctr-search/trial/2016-002066-32/GB.