The FDA has granted breakthrough therapy designation to Hemab Therapeutics’ sutacimig to prevent bleeding in people with the rare disorder Glanzmann thrombasthenia.

Hemab Therapeutics has received breakthrough therapy designation from the US Food and Drug Administration (FDA) for sutacimig as a preventive treatment for bleeding episodes in people living with Glanzmann thrombasthenia.

The designation applies to the use of sutacimig as a prophylactic therapy for the rare inherited disorder, which is characterised by severe and sometimes life-threatening bleeding due to impaired platelet function. There are currently no approved treatments designed to prevent bleeding episodes in this patient population.

Breakthrough therapy designation is intended to accelerate the development and regulatory review of drugs targeting serious or life-threatening conditions where early clinical evidence indicates the potential for substantial improvement over existing therapies.

Hemab said the designation was based on data from the completed Phase 2 multiple ascending dose portion of its Phase 1/2 clinical study, HMB-001-CL101. The results showed consistent reductions in bleeding events among participants, including severe bleeds requiring high-intensity treatments such as recombinant factor VIIa, platelet transfusions, plasma, cryoprecipitate or medical procedures.

Benny Sorensen, chief executive officer of Hemab Therapeutics, said: “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.”

He added: “We look forward to continued collaboration with the FDA, with the goal of bringing the first prophylactic treatment to this patient population.”

Sutacimig is designed to reduce bleeding risk in people with Glanzmann thrombasthenia by targeting underlying mechanisms involved in the disorder. If successful in later-stage trials, the therapy could become the first preventive treatment option for patients, who currently rely on treatments that attempt to control bleeding once it has already started.

According to data from the international Glanzmann’s 360 natural history study, the disease places a heavy burden on patients’ daily lives. Among 117 participants in the study, 88% reported at least one bleeding episode in the previous week, while 65% required a hospital visit related to bleeding within the previous six months.

The condition can significantly affect quality of life. More than 80% of patients reported missing work or school due to bleeding, and over half said the disorder restricted social activities and travel.

Suthesh Sivapalaratnam, consultant haematologist at Barts Health NHS Trust, said: “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.”

He added: “This Breakthrough Therapy Designation is a critical step towards a potentially better therapeutic option for providers and patients who are urgently waiting.”

In addition to breakthrough therapy designation, sutacimig has previously received both fast track and orphan drug designations from the FDA. These regulatory programmes are designed to support the development of treatments for serious conditions and rare diseases.