Timber Pharmaceuticals has received orphan drug designation from the European Commission (EC) for TMB-001 to treat X-linked recessive ichthyosis (XLRI).
Earlier, TMB-001 received orphan drug designation from the EC to treat autosomal recessive congenital ichthyosis (ARCI).
Timber Pharmaceuticals chairman and CEO John Koconis said: “We are pleased to receive an additional orphan drug designation in Europe for the treatment of XLRI as we continue to make steady progress with our global Phase III clinical trial.
“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.
“We believe the targeted delivery of therapies to the epidermis and dermis may be able to minimise systemic absorption and we are committed to bringing a potential new treatment option to this rare disease community.”
Formulated using Timber’s IPEG delivery system, TMB-001 is a topical isotretinoin being developed to treat moderate to severe forms of CI, a group of rare genetic keratinization disorders that cause dry, thickened, and scaling skin.
Cutaneous manifestations in patients with ARCI and XLRI can include large, dark scaling throughout the body.
This is expected to affect several aspects of the quality of life of a person.
Timber is evaluating TMB-001’s pharmacokinetics, efficacy and safety in the ongoing pivotal Phase III ASCEND clinical trial at research centers in Canada, the US, Italy, France, and Germany.
The study will enroll nearly 142 patients aged six years and older with moderate to severe CI.