Filsuvez

The approval was based on data from the randomized, double-blind, placebo-controlled phase 3 EASE study (ClinicalTrials.gov Identifier: NCT03068780), which evaluated the efficacy and safety of Filsuvez in adults and pediatric patients 6 months of age and older with JEB and DEB. Study participants were randomly assigned to receive Filsuvez (n=109) or placebo (n=114) and instructed to apply approximately 1mm (0.04 inch) to all affected areas at each dressing change (every 1 to 4 days) for 90 days.

At randomization, 1 wound was selected by the investigator as the target wound for the evaluation of the primary efficacy endpoint. The target wound was defined as a partial-thickness wound of 10–50 cm² in surface area and present for 21 days to 9 months prior to screening. The primary endpoint was the proportion of patients with first complete closure of the target wound by day 45 of the 90-day double-blind phase of the study, based on clinical assessment by the investigator.

Findings showed 41.3% of patients treated with Filsuvez had first complete closure of target wound within 45 days vs 28.9% of patients treated with placebo gel. Moreover, based on EB subtype, the following proportion of patients treated with Filsuvez had first complete closure of target wound within 45 days vs those in the placebo group, respectively:

• Recessive DEB (n=175): 44% vs 26.2%
• Dominant DEB (n=20): 50% vs 50%
• JEB (n=26): 18.2% vs 26.7%

Results also showed 50.5% of patients treated with Filsuvez achieved first complete closure of target wound within 90 days compared with 43.9% of those who received placebo.