European Commission Approves Amryt’s Filsuvez for the treatment of Dystrophic and Junctional EB

The European Commission approved Amryt’s Filsuvez in the European Union for the treatment of partial thickness wounds associated with dystrophic and junctional epidermolysis bullosa in patients 6 months and older.

Photo: Joe Wiley, CEO of Amryt Pharma

Filsuvez is the first treatment approved in any market to treat epidermolysis bullosa (EB), a rare and devastating group of hereditary disorders of the skin, mucous membranes, and internal epithelial linings characterized by extreme skin fragility and blister development. Patients with severe forms of EB suffer from severe, chronic blistering, ulceration and scarring of the skin, mutilating scarring of the hands and feet, joint contractures, strictures of the esophagus and mucous membranes, a high risk of developing aggressive squamous cell carcinomas, infections, and risk of premature death.

The centralized marketing authorization will be valid in all EU member states as well as in Iceland, Liechtenstein, and Norway.

“The EC approval of Filsuvez in Europe is a major positive development for European patients who suffer from this debilitating condition,” said Joe Wiley, CEO of Amryt Pharma. “ Filsuvez will be our fourth commercial product for rare diseases. We have in place the team, financial flexibility, and global infrastructure to bring it to market swiftly and to execute our significant growth plans.”

European Commission approval of Filsuvez (Oleogel-S10) is supported by phase 3 data from the EASE trial which was the largest ever global trial conducted in patients with EB, performed across 58 sites in 28 countries. It comprised a 3-month double-blind randomized controlled phase followed by a 24-month open-label, single-arm phase. Patients with dystrophic and junctional EB target wounds of between 10 and 50 cm² 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, all entered the open label safety follow up phase.

The EC approval follows a Complete Response Letter issued by the U.S. Food and Drug Administration in February asking the company to submit additional confirmatory evidence of the effectiveness of Oleogel-S10 in EB.

Along with announcing EC approval, Amryt issued notice that it plans to submit a Formal Dispute Resolution Request (FDRR) for its New Drug Application (NDA) for Oleogel-S10. The company said it recently had a Type A meeting with the FDA to discuss the issues raised in the CRL as it seeks to resolve scientific and/or medical disputes that cannot be resolved at the division level.

“The Formal Dispute Resolution process provides an opportunity to raise our scientific disagreement within CDER,” said Wiley. “We believe this represents the right approach toward approval in the United States for Oleogel-S10, as we work toward bringing this therapy to EB sufferers in desperate need.”

Author: Rare Daily Staff