Rare Skin Disorder Therapy Granted Orphan Drug Designation

TWi Pharmaceuticals announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to AC-201 for the treatment of epidermolysis bullosa (EB).

AC-201, a first-in-class small molecule, has shown the ability to inhibit the production and activity of caspase-1 and the cytokine Interleukin-1Beta (IL-1Beta), and to down-regulate IL-1Beta receptors. Inhibition of IL-1Beta signaling has been demonstrated to be effective in treating a variety of diseases including arthritis, gout, and diabetes mellitus.

Currently, TWi Biotechnology holds an Investigational New Drug (IND) application for AC-201 to control blood glucose in patients with type 2 diabetes, and another IND to treat gout in patients under taking urate-lowering therapy. AC-201 has been studied in several human clinical trials including three Phase 2 trials for up to 6 months treatment period and has demonstrated a satisfactory safety profile.

For more information visit TWiPharma.com.

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