The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for Hemab Therapeutics’ drug sutacimig for the prevention of bleeding episodes in patients with Glanzmann thrombasthenia (GT).
GT is a severe bleeding disorder marked by debilitating, sometimes life-threatening bleeding episodes. Bleeding episodes significantly impact patients’ mental health and quality of life, with more than 80% having missed work or school, and 50% facing limitations in attending social events, with 50% experiencing restrictions in travel. To date, there are no effective prophylactic treatment options for GT.
“We are proud to announce this Breakthrough Therapy Designation, which recognizes both the potential for sutacimig to address a significant unmet need, and the urgency of bringing new treatment options to people living with Glanzmann thrombasthenia who have been historically underserved,” said Benny Sorensen, chief executive officer of Hemab.
“We look forward to continued collaboration with the FDA, with the goal of bringing the first prophylactic treatment to this patient population.”
“In people living with Glanzmann thrombasthenia, the impact of frequent bleeding can be life-altering and have an immense impact on quality of life; it results in lost days at school or work and psychological stress. The current therapies inadequately control bleeding let alone prevent it; the unmet need for people living with Glanzmann thrombasthenia is clear,” said Suthesh Sivapalaratnam, consultant haematologist at Barts Health NHS Trust and associate professor at Queen Mary University of London.
“This Breakthrough Therapy Designation is a critical step towards a potentially better therapeutic option for providers and patients who are urgently waiting.”
BTD was granted based on a data set, including positive results from the completed phase 2 multiple ascending dose portion of Hemab’s phase 1/2 clinical trial of sutacimig (HMB-001-CL101). The data showed consistent and clinically meaningful reductions in bleeding, including the most severe bleeding events requiring high-intensity treatments (defined as those requiring recombinant Factor VIIa, platelet transfusions, plasma, cryoprecipitate, or medical procedures). Sutacimig has also received Fast Track Designation and Orphan Drug Designation from the FDA.