The Food and Drug Administration (FDA) has granted Orphan Drug designation to TMB-003 (Timber Pharmaceuticals) for the treatment of systemic sclerosis.

Systemic sclerosis is a rare, chronic autoimmune disorder characterized by inflammation and thickening of the skin and other connective tissues from excessive collagen deposition. TMB-003 is a locally delivered formulation of sitaxsentan, a highly selective endothelin receptor antagonist. The Company believes that a topical or subcutaneous formulation of sitaxsentan has the potential to reduce collagen while addressing the systemic adverse effects associated with oral administration.

“Currently there is no FDA approved treatment for any cutaneous symptoms in scleroderma,” said John Koconis, CEO of Timber. “We are pleased to receive Orphan Drug designation for our investigational treatment and look forward to advancing into clinical stage research.” The Company expects to submit an Investigational New Drug application to the FDA in 2022.

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals. 

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Timber Pharmaceuticals receives Orphan Drug designation from U.S. FDA for TMB-003 for the treatment of systemic sclerosis. [press release]. Woodcliff Lake, NJ: Timber Pharmaceuticals; January 12, 2021.