European Commission grants second orphan drug designation for TMB-001

The European Commission has granted orphan drug designation for TMB-001 for the treatment of X-linked recessive ichthyosis, Timber Pharmaceuticals announced in a press release.

This will be the second such designation for TMB-001, which was previously granted orphan drug designation for the treatment of autosomal recessive congenital ichthyosis.

“These orphan drug designations underscore the significant unmet needs in congenital ichthyosis (CI), which can lead to a limited range of motion, chronic itching, an inability to sweat normally, high risk of secondary infections, and impaired eyesight or hearing,” John Koconis, chairman and CEO of Timber, said in the release.

TMB-001, a topical formulation of 0.05% isotretinoin, is currently being assessed in the ongoing, pivotal phase 3 ASCEND trial. The study will enroll approximately 142 patients in the United States, Canada, Italy, France and Germany, aged 6 years and older with moderate to severe congenital ichthyosis. Koconis reports that “steady progress” is being made in the study.

“We believe the targeted delivery of therapies to the epidermis and dermis may be able to minimize systemic absorption and we are committed to bringing a potential new treatment option to this rare disease community,” Koconis said.