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news.TWi Biotechnology has received the Orphan Drug designation from the US food and Drug Administration (FDA) for AC-201, a first-in-class, small molecule, to treat epidermolysis bullosa (EB).
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AC-201 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 shown effectiveness in treating several diseases including arthritis, gout and diabetes mellitus (DM).
The active ingredient of AC-201 has been approved to treat patients with chronic rheumatic diseases in France and subsequently in other EU and Middle Eastern countries, such as Spain and Italy.
Commenting on the designation, TWi Biotechnology president Dr Calvin Chen said: "It fulfills the mission of our new drug business for meeting unmet medical needs.
"Current treatments of EB are focusing in relief of symptoms such as itching and pain and wound care to prevent infection, which are expensive such as artificial skin and may require frequent visiting to hospitals.
"In contrast, the topical formulation of AC-201 is developed to prevent or reduce the blisters that, if success, may alleviate the suffering of the patients and their families from this deliberating disease."
According to the estimates, about 30,000 patients in US, and a few hundreds of children in Taiwan suffered from the deliberating disease, which is also known as ‘Butterfly Children’ in the US and ‘Bubble Dragan Children’ in Taiwan.