Epidermolysis bullosa gene therapies wait in the wings

By the end of this year, Amryt’s Filsuvez could become the first approved treatment for the rare skin disorder epidermolysis bullosa. Despite mixed pivotal data, regulators in the US and Europe might be swayed by the lack of available treatments.

More durable options could come with gene therapies, and phase 3 studies of these will start to report later this year. However, gene therapy procedures can be complex, and Amryt will hope that the convenience of Filsuvez, a topical gel that can be applied at home, could make it the first-line treatment of choice.