TAIPEI, March 10, 2015 /PRNewswire/ — TWi Pharmaceuticals, Inc. today announced that its fully owned subsidiary, TWi Biotechnology, Inc., has received the designation of Rare Disease Drug by Taiwan FDA for use of AC-203 to treat Epidermolysis Bullosa Simplex (EBS), and is eligible for applying for coverage under National Health Insurance Administration (NHIA). TWi Biotechnology has developed proprietary formulations to be used topically for the indication of EBS and will plan for further development and product launch according to the designation.
"We are very pleased with the TFDA’s approval of the rare disease drug designation for treating epidermolysis bullosa simplex. This is a major milestone of AC-203, which has obtained the orphan drug designation from U.S. FDA just several months ago. In addition, this fulfills the mission of our new drug business for developing proprietary formulations and meeting unmet medical needs," said Dr. Calvin C. Chen, President of TWi Biotechnology. "Epidermolysis Bullosa, or EB, is a very rare genetic connective tissue disorder that affects several hundred of children in Taiwan suffering from this disease; patients with EB are known as ‘Bubble Dragon Children’ in Taiwan or ‘Butterfly Children’ in the United States due to their extremely fragile skin that blisters and tears from friction or trauma. We hope this rare disease drug designation can not only help the patients in Taiwan but also speed the development of topical AC-203 in other places of the world. Current treatments for EBS focus on relief of symptoms such as itching and pain, and wound care to prevent infection, which include expensive treatments such as artificial skin and may require frequent visits to hospitals. In contrast, the topical formulation of AC-203 is developed to prevent or reduce the blisters that, if successful, may alleviate the suffering of the patients and their families from this deliberating disease."
AC-203 is the topical formulation of a first-in-class small molecule known as AC-201 which 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 (DM). The active ingredient of AC-201 has been approved for treating patients with chronic rheumatic diseases in France and subsequently in other EU and Middle Eastern countries such as Spain and Italy since mid-1990.
TWi Biotechnology holds two US INDs for AC-201 – one for controlling blood glucose in patients with type II diabetes and the other for treating gout in patients undertaking urate-lowering therapy. In addition to the good safety record of its active ingredient used in treating another chronic disease, AC-201 has undergone testing in several human clinical trials including 3 phase II trials for up to 6 months treatment period with a satisfactory safety profile demonstrated.
About Epidermolysis Bullosa (EB)
EB is caused by mutations in the DNA code that make up genes. Genes are responsible for making and expressing proteins. EB can result from one genetic mutation in any one of the 18 genes that have been found to cause the disorder. These mutations, or errors in the genetic code, may not allow the affected gene to produce a specific protein, or it may result in the incorrect formation of that protein. Either way, the mutation disrupts the correct function of the protein, thus resulting in extremely fragile skin, and other manifestations of EB including: anemia, cardiomyopathy, syndactyly (fusion of the fingers and toes), renal insufficiency, dysphagia (difficulty swallowing), malnourishment, cancer, constipation, osteoporosis, muscular dystrophy and pyloric atresia.
About TWi Biotechnology, Inc.
TWi Biotechnology, Inc., a fully owned subsidiary of TWi Pharmaceuticals, Inc., is a leading clinical stage biopharmaceutical company based in Taipei, Taiwan, specializing in the development of innovative new drugs for unmet medical needs, especially in the diseases associated with innate immunity. The company is building its product pipeline through in-licensing and internal research. TWi Biotechnology’s product development pipeline includes three drug candidates for treating type II diabetes, retinopathy, arthritis, and immunodermatology diseases.
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