Vaderis Therapeutics AG (Vaderis), a clinical stage biotechnology company focusing on treatments for rare diseases associated with vascular malformations, today announces that the US Food and Drug Administration (FDA) has designated the allosteric AKT-inhibitor VAD044 a Fast Track product for the treatment of Hereditary Hemorrhagic Telangiectasia (HHT).
Fast Track is an FDA process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The FDA states the purpose of Fast Track to get important new drugs to the patient earlier.
Dr. Hanny Al-Samkari, the Peggy S. Blitz Endowed Chair in Hematology/Oncology at Massachusetts General Hospital and Associate Professor of Medicine at Harvard Medical School (USA) commented, “The FDA’s decision to designate VAD044 as a Fast Track product for the treatment of HHT underscores its potential to be the first ever approved treatment for this debilitating genetic disease.”
HHT, an Orphan Disease, is the second most common inherited bleeding disorder in the world frequently causing severe disease burden, reduced life expectancy and impaired Quality of Life. Despite this, there remains no approved treatment for HHT anywhere in the world. Vaderis is developing VAD044, an oral, once-daily allosteric AKT-inhibitor, the first novel therapy intended specifically for the treatment of HHT.
Nicholas Benedict, CEO and co-Founder of Vaderis Therapeutics commented, “The excitement surrounding the results of the initial 12-week double-blind part of this trial is amplified by the continued improvements experienced by patients through 6 months. Excellent collaboration with patient and physician organisations such as CureHHT has been a cornerstone of successful implementation of this groundbreaking trial which was achieved in a much shorter timeframe than planned. Vaderis is currently interacting with major health authorities to plan the pivotal phase of development for VAD044 in HHT.”
