Research & Development
OBI Pharma Inc wins US FDA's orphan drug designation for OBI-3424 for Acute Lymphoblastic Leukemia
20 September 2018 -

Taiwan biopharma company OBI Pharma Inc (TPEx:4174) reported on Wednesday the receipt of the US Food and Drug Administration's (FDA) orphan drug designation (ODD) for OBI-3424 for the treatment of Acute Lymphoblastic Leukemia (ALL),also known as Acute Lymphocytic Leukemia.

The company said OBI-3424 is a first-in-class novel small-molecule prodrug that selectively targets cancers overexpressing the enzyme aldo-keto reductase 1C3 (AKR1C3), and selectively releases a potent DNA alkylating agent in the presence of the AKR1C3 enzyme. This selective mode of activation distinguishes OBI-3424 from traditional alkylating agents, such as cyclophosphamide and ifosfamide, which are non-selective.

This US FDA's orphan drug designation is reportedly the second for OBI-3424. In July, OBI-3424 was granted orphan drug status for Hepatocellular Carcinoma (HCC).

Following the US FDA's orphan drug designations, a Phase 1/2 study of OBI-3424 in patients with solid tumors, including HCC and castrate-resistant prostate cancer (CRPC), has commenced enrolment at the University of Texas M.D. Anderson Cancer Center.

Acute Lymphoblastic Leukemia (ALL) is a rare blood cancer affecting the maturation of B-cell and T-cell lymphoblasts from progenitor cells. The disease affects primarily children and the remission rate for paediatric ALL is approximately 90%, with overall survival around 60-70% in recent years.

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