RARE Daily

Amryt Reports Positive Results from Pivotal Trial of Therapy for Epidermolysis Bullosa

September 9, 2020

Rare Daily Staff

Amryt reported that the pivotal phase 3 EASE trial of its experimental therapy Filsuvez for the treatment of dystrophic and junctional epidermolysis bullosa met the primary endpoint of speed of wound closure with statistical significance.

Epidermolysis bullosa (EB) can cause skin to blister and tear from the slightest friction or trauma and can, in some cases, cause blistering and erosion of the epithelial lining of internal organs. EB is chronic, potentially disfiguring and in some cases fatal. There are currently no approved treatments.

Filsuvez (formerly called AP101) is a topical gene therapy that is designed to deliver a functional copy of the collagen gene directly to the skin. In the phase 3 clinical trial, the largest ever global phase 3 study for EB.

“This positive outcome of the phase 3 EASE trial marks another significant milestone for Amryt as we seek approval for Filsuvez and represents a potentially important advancement for patients and families living with this rare and distressing disorder,” said Joe Wiley, CEO of Amryt.

The EASE trial is the largest ever global phase 3 trial conducted in patients with EB, performed across 58 sites in 28 countries. It comprises a three-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 50 cm2 in size that were present for greater than 21 days and less than 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.

A total of 223 patients were enrolled into the trial including 156 pediatric patients.  Of those that completed the double-blind phase, all entered the open label safety follow up phase.

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. Although the company did not break out the data, it said the primary endpoint was achieved with statistical significance.

Amryt said the results represent the first successful phase 3 top line readout in EB, and the fourth time Filsuvez has demonstrated accelerated wound healing in a phase 3 trial.

While the key secondary endpoints did not achieve statistical significance, a number of favorable differences were observed. In addition, substantial further secondary endpoint data is expected and will be analyzed over the coming weeks.

Amryt will evaluate and analyze the full data set from the trial and will present results at an upcoming scientific symposium. The company intends to complete the submission of its rolling New Drug Application to the U.S. Food and Drug Administration and request priority review. Filsuvez has previously received Fast Track and Rare Pediatric Disease designations from the FDA. Amryt also intends to pursue an accelerated assessment in the EU. Regulatory submissions in the US and the EU are expected to be filed by the end of the first quarter of 2021.

Amryt Currently markets Juxtapid, a treatment for adults with the rare cholesterol disorder homozygous familial hypercholesterolemia (HoFH), and Myalept, an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.

Photo: Joe Wiley, CEO of Amryt


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