Q BioMed Inc, a biotech company, announced that the US Food and Drug Administration's Office of Orphan Products Development has granted Orphan Drug Designation to Uttroside-B, a small molecule chemotherapeutic for the treatment of hepatocellular carcinoma (HCC), the most common form of liver cancer. In preclinical studies, Uttroside-B was up to 10-times more potent against HCC cells than Sorafinib, the standard of care drug at the time.As an Orphan Drug, Uttroside-B may benefit from a seven-year market exclusively following marketing approval, grant funding for clinical trials that contribute to marketing approval, protocol assistance, and tax credits. Preclinical testing is now underway to support an US FDA Investigational New Drug (IND) application expected this year. Q BioMed has the exclusive rights to the technology through an agreement with the Rajiv Gandhi Centre for Biotechnology, an autonomous Institute under the Department of Biotechnology, Government of India and the Oklahoma Medical Research Foundation."Orphan Drug Designation gives our Uttroside-B program a substantial boost, and we expect it will significantly accelerate development and reduce costs. We are pleased that the US FDA recognizes the urgent need for effective treatments for HCC, and that the agency sees the potential of Uttroside-B to address this difficult to treat cancer," stated Q BioMed CEO Denis Corin.With very limited approved first-line pharmaceutical therapies for HCC available today, challenges include drug resistance, adverse side effects, and high costs. An estimated 700,000 people are diagnosed with HCC each year, with the global market for liver cancer drugs expected to grow to US $3.9 billion by 2027.
