CHMP Recommends Approval of Amryt’s Filsuvez for Dystrophic and Junctional EB

The European Medicines Agency’s Committee for Medicinal Products for Human Use has adopted a positive opinion, recommending the approval of Amryt Pharma’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

Epidermolysis bullosa (EB) is a rare 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. There is currently no approved treatment and Amryt estimates the global market opportunity to be in excess of $1.0 billion.

Based on the CHMP recommendation a decision by the European Commission (EC) is expected on the Filsuvez application within 67 days, which would be valid in all EU member states as well as in Iceland, Liechtenstein, and Norway. The Medicines and Healthcare products Regulatory Agency (MHRA) in the U.K. is expected to grant authorization within the same time period.

The CHMP positive opinion 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 50cm2 in size that were present for more 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. 223 patients were enrolled into the trial including 156 pediatric patients. Of those that completed the double-blind phase, 100 percent entered the open label safety follow up phase.

As a result of the CHMP positive opinion, the EMA Contingent Value Right (CVR) issued to Amryt shareholders and option holders prior to the acquisition of Aegerion will now become payable.

“The CHMP recommendation for approval of Filsuvez in Europe is the most significant milestone in Amryt’s history and represents a major positive development for European patients that suffer from this debilitating condition. This is the culmination of years of hard work from all the Amryt team,” said Joe Wiley, CEO of Amryt Pharma. “Filsuvez would be our fourth commercial product and we already have in place the team, financial flexibility, systems and global infrastructure to bring it to market and to execute our significant growth plan.”

Author: Rare Daily Staff