News Feature | October 27, 2014

FDA Awards Orphan Status To TWi Pharma's Rare Skin Disease Drug

By Estel Grace Masangkay

TWi Pharmaceuticals reported that its subsidiary TWi Biotechnology has been awarded Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its drug AC-201 as treatment for rare skin disease epidermolysis bullosa (EB).

AC-201 is a first-in-class small molecule designed to inhibit the production and activity of caspase-1 and the cytokine Interleukin-1Beta (IL-1Beta) as well as to down-regulate IL-1Beta receptors. IL-1Beta inhibition has also been a potential treatment for other diseases, such as gout, diabetes mellitus, and arthritis. AC-201’s active ingredient has received approval in EU and other countries as treatment for chronic rheumatic diseases. The company has also received two INDs for the drug in the U.S. to investigate the drug as therapy for diabetes and treatment for gout.

Dr. Calvin C. Chen, President of TWi Biotechnology, said, “We are very pleased with the approval of orphan drug designation for treating epidermolysis bullosa… We hope this orphan drug designation can result in a speedy development of topical AC-201. Current treatments of EB are focusing in relief of symptoms… In contrast, the topical formulation of AC-201 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.”

A rare connective tissue disorder, Epidermolysis Bullosa (EB) causes extremely fragile skin, and can also be responsible for causing anemia, cardiomyopathy, dysphagia, malnourishment, cancer, and osteoporosis, among others. EB affects 1 out of every 50,000 births. There are an estimated 30,000 patients suffering from Epidermolysis Bullosa in the U.S.

The company said that it plans to progress to clinical trials to investigate the topical formulation of AC-201 as treatment for EB.

Earlier this year TWi Pharmaceuticals announced its acquisition of Hainan Visum Pharmaceutical, a specialist pharmaceutical company in China. The company said it expects a series of mergers and acquisitions on the international level as a result of the transaction.