Telormedix, a clinical stage biopharmaceutical company focused on TLR7 agonists in the treatment of cancer and infectious diseases, has obtained the orphan drug designation to Vesimune for the treatment of carcinoma in situ (CIS) in the bladder, by the U.S. Food and Drug Administration (FDA).
Vesimune (TMX-101), is a proprietary targeted small molecule for the treatment of superficial bladder cancer. The active ingredient is a known immunomodulatory molecule with a favourable safety profile and a demonstrated clinical efficacy in oncological and viral diseases. It is Telormedix’ lead product, a TLR-7 agonist, that has successfully completed a Phase II trial in CIS of the bladder. The product is a unique sterile liquid formulation of a marketed immune modulatory compound, designed on innovative technology principles to carrier drug delivery systems in order to increase solubility, bio-adhesiveness and stability. These properties mean that the product can be used in therapeutic settings that the original product could not.
The FDA grants orphan drug status to products for rare diseases and defines a rare disease as one with a prevalence of less than 200,000 cases in the USA.