Argo Biopharmaceutical Co., Ltd. has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to its novel siRNA therapy BW-20805, for the treatment of hereditary angioedema (HAE).
HAE is a rare genetic condition that causes sudden and unpredictable swelling in different parts of the body. In severe cases, it can affect the throat and become life-threatening, with a mortality rate of up to 40%. HAE affects about 1.5 people per 100,000 worldwide. Current prophylactic treatments are limited by frequent dosing, highlighting the need for long-acting, preventive therapies.
BW-20805 is an investigational siRNA therapy that targets and significantly inhibits prekallikrein (PKK), a well-validated target for HAE treatment, offering the possibility of prevention of HAE attacks with a long-term effect. Argo Biopharma is currently conducting a global phase II study in adult HAE patients. The Company anticipates primary completion of the phase II study in the second half of 2026, followed by plans for a global phase III study.
“Receiving FTD from the FDA highlights the significant unmet medical need for patients living with HAE and underscores the potential of BW-20805 as a novel therapeutic option,” said Dongxu Shu, co-founder and chief executive officer of Argo Biopharma.
“We have generated a robust body of clinical evidence supporting BW-20805’s potential, including recent open-label study results presented at the AAAAI Annual Meeting, which demonstrated that BW-20805 provides remarkable plasma PKK reduction and meaningful time-normalized HAE attack rate reduction. We look forward to advancing the clinical development of BW-20805 and bringing a potentially long-acting treatment option to patients as effectively as possible.”