Timber Pharmaceuticals has announced the European Commission (EC) granted orphan drug designation for TMB-001 for the treatment of X-linked recessive ichthyosis (XLRI) on February 15, 2023.
The EC previously granted orphan drug designation for TMB-001 for the treatment of autosomal recessive congenital ichthyosis (ARCI).
Timber Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the development and commercialisation of treatments for rare and orphan dermatologic diseases.
“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 3 clinical trial,” said John Koconis, Chairman and Chief Executive Officer of Timber. “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.”
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 3 clinical trial
TMB-001 is a topical isotretinoin, formulated using the company’s patented IPEG delivery system, in development for the treatment of moderate to severe forms of CI. CI is a group of rare genetic keratinization disorders that lead to dry, thickened, and scaling skin. In patients with ARCI and XLRI, cutaneous manifestations can include large, dark scaling throughout the body, which affect multiple aspects of the person's quality of life.
Timber is assessing the efficacy, pharmacokinetics and safety of TMB-001 (0.05% isotretinoin) in the ongoing pivotal Phase 3 ASCEND clinical trial at leading research centres in the US., Canada, Italy, France, and Germany. The study will enrol approximately 142 patients ages six years and above with moderate to severe CI.
Orphan drug designation is available for treatments for rare diseases that are life-threatening or chronically debilitating that affect fewer than five in 10,000 people across the European Union (EU). Medicines that are granted orphan drug designation by the EC qualify for financial and regulatory incentives including protocol assistance at reduced fees during product development, access to centralised marketing authorisation, and 10 years of market exclusivity in the EU after product approval.