HUYA Bioscience International (HUYA), Founder, CEO & Executive Chair, Dr. Mireille Gillings today announced that the Ministry of Health, Labour and Welfare (MHLW) granted HBI-8000 orphan drug designation in Japan for peripheral T-cell lymphoma (PTCL). HBI-8000 is a novel class I-selective oral histone deacetylase (HDAC) inhibitor with immunomodulatory effects regulating antitumor activity, as well as repression of genes associated with drug resistance. The product is currently completing a Phase 1 open-label, dose escalation trial in Japan to evaluate the safety and pharmacokinetics of HBI-8000 in Japanese patients with non‑Hodgkin's lymphoma (NHL). The orphan drug designation was based on the estimated size of the Japanese PTCL patient population, the non-clinical and clinical studies as well as the clinical development plan in Japan.
"The breadth of activity of HBI-8000 is now emerging strongly as more studies are conducted. The orphan drug designation for PTCL is an important demonstration of efficacy to combat a devastating unmet need," said Dr. Mireille Gillings, Founder, CEO & Executive Chair of HUYA. "With the orphan drug designation, HBI-8000 should benefit from a priority review for marketing authorization and a 10-year market exclusivity as well as financial incentives."
Under the Tripartite Cooperation Treaty between China, South Korea and Japan and given the recent approval of the drug for the treatment of PTCL in China, HUYA launched development of HBI-8000 in Japan for NHL. The Company plans to begin a Phase 2 registration trial in 2016 based on the Pharmaceutical and Medical Devices Agency's (PMDA) acceptance of HUYA's accelerated development strategy for Japan.