Hemab Therapeutics Receives Breakthrough Therapy Designation for Sutacimig in Glanzmann Thrombasthenia
Friday, March 06, 2026
Hemab Therapeutics has received Breakthrough Therapy Designation (BTD) from the US Food and Drug Administration (FDA) for sutacimig, a potential treatment aimed at preventing bleeding episodes in patients with Glanzmann thrombasthenia (GT).
Glanzmann thrombasthenia is a rare inherited bleeding disorder characterised by recurrent and sometimes life-threatening bleeding episodes. Data from the international Glanzmann’s 360 natural history study highlighted the significant burden of the disease. Among 117 participants, most reported at least one bleeding episode during the previous week, and many required hospital visits related to bleeding within six months. The condition also affects daily life, with many patients missing school or work and experiencing restrictions on social activities and travel. At present, there are no effective preventive treatments available for GT.
Sutacimig, previously known as HMB-001, is a subcutaneously administered bispecific antibody. It is designed to bind to endogenous Factor VIIa with one arm and to TLT-1 on activated platelets with the other. This mechanism aims to stabilise Factor VIIa and direct it to the surface of activated platelets, helping to support the formation of a haemostatic plug and reduce bleeding. The therapy is being developed as a first-in-class prophylactic treatment for Glanzmann thrombasthenia, with potential applications in other bleeding disorders.
The company is developing the therapy as a prophylactic option for people living with serious coagulation disorders that currently have limited treatment choices.
The designation highlights the potential of sutacimig to address a significant unmet medical need in patients with GT, a rare and severe bleeding disorder. Breakthrough Therapy Designation is granted by the FDA to speed up the development and review of medicines intended for serious or life-threatening conditions when early clinical evidence suggests they may offer substantial improvement over existing treatments.
The decision was supported by a comprehensive set of clinical data, including results from the completed Phase 2 multiple ascending dose portion of Hemab’s Phase 1/2 clinical trial of sutacimig. The study showed consistent and clinically meaningful reductions in bleeding episodes, including severe events that require intensive treatments such as recombinant Factor VIIa, platelet transfusions, plasma, cryoprecipitate or medical procedures.
Sutacimig has previously received both Fast Track Designation and Orphan Drug Designation from the FDA, reflecting the need for improved therapies in this patient population.
In addition to the FDA designations, sutacimig has received designation under the Innovative Licensing and Access Pathway (ILAP) from the UK Medicines and Healthcare products Regulatory Agency and orphan medicinal product status in the European Union for the treatment of Glanzmann thrombasthenia.
Source: prnewswire.com
