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."