Amryt Pharma reported top line results from its pivotal Phase 3 EASE trial of Filsuvez (previously AP101 /Oleogel-S10) for the treatment of dystrophic and junctional Epidermolysis Bullosa (EB). The primary endpoint of the trial was met with statistical significance and Filsuvez was well tolerated.
EASE comprised a 3-month double-blind randomized controlled phase followed by a 24-month open-label, single-arm phase. Patients with EB target wounds of between 10 and 50cm2 in size that were present for > 21 days and < 9 months were randomized in the double-blind phase to study treatment in a 1:1 ratio and wound dressings applied according to standard of care. 223 patients were enrolled into the trial including 156 pediatric patients. Of those that completed the double-blind phase, 100% entered the open label safety follow up phase. The trial was conducted across 58 sites in 28 countries
The primary endpoint of the trial was to compare the efficacy of Filsuvez versus control gel according to the proportion of patients with complete closure of the target wound within 45 days of treatment. The primary endpoint was achieved with statistical significance. The key secondary endpoint, time to first complete closure of the EB target wound as evidenced by clinical assessment until Day 90, did not achieve statistical significance. However, some favorable differences were observed.
Amryt plans to evaluate and analyze the full data set from the trial and will present results at an upcoming scientific symposium.
A rolling New Drug Application (NDA) has been submitted to the US FDA and priority review will be requested. Amryt also intends to pursue an accelerated assessment in the EU.
Filsuvez has received Fast Track Designation and Rare Pediatric Disease Designation from the FDA and has been granted Orphan Drug status in the EU and the US.
Filsuvez (AP101 /Oleogel-S10) is a topical product incorporating a betulin-based active ingredient formulated with sunflower oil. Betulin is abundant in birch bark, and AP101 consists of 10% birch bark extract in 90% sunflower oil.AP101 causes the migration of keratinocytes (cells that regenerate the outer layer of the skin) to the wound site and their differentiation into mature epithelial skin cells, leading to faster wound healing and skin restoration.
About Epidermolysis Bullosa
Epidermolysis Bullosa is a group of rare and genetic disorders of the skin, characterized by skin fragility which can be severe and cause recurrent blistering of the skin in response to minor trauma or friction. It is caused by some of the layers of the skin, in the dermis and epidermis, not sticking together properly. In most cases, the effects of EB are seen at birth or shortly after. The most significant problems in severe forms of EB are the life-long presence of blistering, wound infections and partially chronic wounds resulting in pain, scarring and immobility. Dressing changes to treat these wounds can take several hours each day and are extremely painful, often requiring the use of opioid drugs to manage the pain.