Timber Pharmaceuticals Secures European Orphan Drug Designation

Timber Pharmaceuticals (Nasdaq: TMBR) has received Orphan Drug Designation for TMB-001 for the treatment of X-linked recessive ichthyosis (XLRI) from the European Commission (EC).

This is the company’s second orphan drug designation granted by the EC for TMB-001, which has also received the designation for the treatment of autosomal recessive congenital ichthyosis (ARCI).


  • 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 centralized marketing authorization and 10 years of market exclusivity in the EU after product approval.
  • 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 centers in the U.S., Canada, Italy, France, and Germany.
  • The study will enroll approximately 142 patients, including children over the age of six, with moderate to severe congenital ichthyosis.

Orphan drug designations underscore the significant unmet medical need in congenital ichthyosis, 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.


This communication was produced by Prism MarketView, an affiliate of PCG Advisory Inc., (together “PCG”). PCG is an integrated investor relations, communications and strategic advisory firm. The information contained on this is ‘Paid Advertising’ for purposes of Section 17(b) of the Securities Act of 1933, as amended (together with the rules and regulations there under, the “Securities Act”). “PCG” and its affiliates are compensated by respective clients for publicizing information relating to its client’s securities. For more information in terms of compensation received for services provided by PCG, see the pertinent advertising materials relating to the respective client. By accessing this Site and any pages thereof, you agree to be bound by the Terms of Use and Privacy Policy…

Read Full Disclaimer

Share This Article


About the Author

Timber Pharmaceuticals Secures European Orphan Drug Designation

Editor Prism MarketView