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 Chugai Pharmaceutical Co., Ltd. announced that Dr. Kunihiro Hattori (former senior fellow of Chugai), Dr. Takehisa Kitazawa (deputy head of research division, Chugai), and Dr. Tomoyuki Igawa (head of research division, Chugai), who led the creation of Hemlibra (emicizumab), a treatment for haemophilia A, have been awarded the Lasker-DeBakey Clinical Medical Research Award, one of the world’s most prestigious scientific honours.
Haemophilia A is caused by a deficiency of coagulation Factor VIII, one of the proteins required for normal blood clotting. When activated, Factor VIII serves as a bridge that facilitates the activation of Factor X by activated Factor IX on the surface of activated platelets. The award recognizes the unconventional concept of replacing this function of Factor VIII with a bispecific antibody, which enabled prolonged therapeutic activity, the convenience of subcutaneous administration, and sustained bleed prevention regardless of the presence or absence of Factor VIII inhibitors(antibodies), thereby making a significant contribution to addressing unmet medical needs in haemophilia A.
The Lasker Awards are among the most respected medical research awards in the United States, honouring individuals who have made significant contributions to medical science and public health. They are presented in the fields of basic medical research, clinical medical research, and public service. This marks the second time Japanese researchers have received the award in the clinical medical research category since the inception of the Lasker Awards in 1945, and the first time that three Japanese researchers have been recognized simultaneously in this category.
Emicizumab is an antibody drug created through extensive research conducted by Chugai scientists. Conventional antibody therapeutics typically exert their effects through a “subtractive” approach, inhibiting the activity of disease-related molecules or cells, or eliminating target cells through immune-mediated mechanisms. In contrast, emicizumab became the world’s first antibody medicine to embody an “additive” approach, restoring a missing biological function by endowing the antibody itself with a function normally performed by a different protein. It is also the world’s first recombinant full-length IgG bispecific antibody therapeutic designed to bind two different targets. During its creation, Chugai also developed and applied its proprietary antibody engineering technology, ART-Ig, which enabled commercial-scale manufacturing.
The realization of this treatment was made possible through collaboration with Nara Medical University, which possesses extensive expertise in both basic and clinical research on haemophilia. Emicizumab was licensed out to Roche and Chugai worked together with Roche and Genentech to advance global clinical development and regulatory submissions, ultimately bringing the product to patients as Hemlibra.
Following its approval in the United States in 2017 and in Japan and Europe in 2018, Hemlibra is now approved in more than 120 countries and regions worldwide. An integrated analysis of the phase III HAVEN 1-4 studies demonstrated that, regardless of the presence or absence of factor VIII inhibitors, disease severity, or age, the primary endpoint of the annualized bleeding rate (ABR) for treated bleeds over 24-week intervals throughout the study was 1.4 events per year. Moreover, 70.8% of participants (277/391) experienced zero treated bleeds during Weeks 1-24, and this proportion increased over time to 82.4% (140/170) during Weeks 121-144. No new safety signals were identified, and the most common adverse event was injection-site reactions (27.8%).
In addition to providing sustained bleed prevention regardless of the presence or absence of factor VIII inhibitors, emicizumab offers a new treatment option by not inducing new factor VIII inhibitors and improving convenience through subcutaneous administration and extended dosing intervals. To date, it has been used by more than 30,000 people with haemophilia A worldwide (cumulative global total as of June 30, 2026).
Dr Osamu Okuda, Chugai’s president and CEO, commented, “I am truly delighted that this original concept, inspired by our desire to reduce the burden on people with haemophilia A and their families, together with the innovative drug discovery technologies that brought it to life, has been recognized through this prestigious award. Emicizumab has delivered sustained bleed prevention regardless of the presence or absence of inhibitors, disease severity, or age, while reducing treatment burden through subcutaneous administration once every one to four weeks*, thereby bringing a new everyday life to people with haemophilia A and their families. I would like to extend my heartfelt congratulations to the three researchers and express my profound respect for their creativity, leadership, and perseverance in tackling formidable challenges, as well as for the many individuals whose dedication and contributions to research and development made this achievement possible.
“This scientific breakthrough could not have been realized as Hemlibra and delivered to people with haemophilia A around the world without the collaboration and support of healthcare professionals, including those at Nara Medical University, as well as our partners at Roche and Genentech. I would also like to express my sincere gratitude to all those who have supported this endeavour over many years.
“Looking ahead, we will continue to strengthen our proprietary technologies and scientific capabilities while promoting collaboration with diverse partners. Through these efforts, we remain committed to delivering innovative drugs and services to patients around the world.”
Remarks from Laureates
Dr. Kunihiro Hattori: Contribution: Originated the concept of a bispecific antibody based on expertise in blood coagulation and antibodies, and led the research project, said: “I am deeply honoured that our collective efforts, together with many researchers and clinicians, dedicated to advancing science for society and for patients, have been recognized through this award. I believe our continuing mission is to ensure that this treatment reaches patients around the world who can benefit from it.”
Dr. Takehisa Kitazawa: Contribution: Led pharmacology and biology research, advancing the discovery of the candidate antibody and the demonstration of factor VIII-mimetic activity, said: “It has been a privilege to contribute to the creation of emicizumab through collaboration with numerous healthcare professionals and fellow researchers. I would also like to extend my heartfelt gratitude to the people with haemophilia A and their families who participated in the clinical studies. I hope this recognition will inspire further innovation, and I remain committed to advancing the next generation of scientific breakthroughs.”
Dr. Tomoyuki Igawa: Contribution: Led the design of emicizumab and the establishment of technologies for the efficient manufacturing of bispecific antibodies, said: “The desire to lessen the burden on patients and their families while helping them lead fuller lives has motivated us to overcome many scientific challenges. I am truly honoured that the three of us have received such a prestigious award together. We will continue to pursue innovative drugs that make a meaningful difference in people’s lives through scientific excellence and creative thinking.”
Haemophilia is primarily classified into two types, haemophilia A and haemophilia B. Haemophilia A is a bleeding disorder caused by a deficiency of a protein called coagulation factor VIII (FVIII), which is essential for normal blood clotting. As a result, people with haemophilia A have difficulty forming blood clots. Haemophilia A can be either congenital, caused by an inherited genetic abnormality, or acquired, which develops later in life. Congenital haemophilia A predominantly affects males and occurs in approximately one in 5,000 male births. Occurrence in females is extremely rare because congenital haemophilia A is an X-linked recessive disorder.
Symptoms associated with bleeding in haemophilia A vary, but a characteristic feature of the disease is the frequent occurrence of internal bleeding that is not externally visible. Internal bleeding can lead to the formation of hematomas, or localized collections of blood, which may compress surrounding nerves and blood vessels, causing pain and functional impairment. Bleeding commonly occurs in joints such as the elbows, knees, and ankles, as well as in muscles, often resulting in swelling, warmth, and severe pain. In patients with severe haemophilia A, bleeding into joints and muscles can occur even during normal daily activities. Repeated joint bleeding may lead to progressive joint damage, significantly affecting quality of life.
Since around 2000, prophylactic treatment with regular replacement of the missing factor VIII has become widely adopted. However, this approach requires intravenous infusions as frequently as once to several times per week. In addition, people with haemophilia A who developed an immune response to factor VIII, a non-self protein, resulting in the formation of factor VIII inhibitors, faced significant treatment challenges due to the limited treatment options available to them.
Established in 1945 by Albert and Mary Lasker. Through its internationally renowned Lasker Awards, educational initiatives, and public advocacy, the Foundation raises awareness of the power of biomedical science to save and improve human lives. Through these efforts, the Foundation advocates for support for biomedical research, with the goal of advancing the prevention and treatment of disease and disability.
Renowned as America’s preeminent biomedical research prize, 360 laureates have received Lasker Medical Research Awards since 1945. Over these years, 101 Lasker Laureates have also received the Nobel Prize, including 28 in the last two decades. The laureates are selected by an international jury chaired by Joseph L. Goldstein, who received both the Lasker Award for Basic Medical Research and the Nobel Prize in Physiology or Medicine in 1985.
The Department of Paediatrics at Nara Medical University has long been a pioneer in haemophilia care in Japan and has advanced both basic and clinical research in the field over many years.
Since 2003, Chugai has conducted collaborative research with the university. This collaboration has generated numerous achievements, including the establishment of coagulation assay systems for evaluating emicizumab.
In addition, the university made significant contributions to the clinical development of emicizumab, including its early clinical studies in Japan. Furthermore, during global clinical development, the network of international researchers and haemophilia specialists cultivated over many years by the Department of Paediatrics at Nara Medical University served as a major asset for Chugai as it entered a new therapeutic area.
Chugai Pharmaceutical Co., Ltd., headquartered in Tokyo, is a research-based pharmaceutical company with world-class drug discovery capabilities, including proprietary antibody engineering technologies. Chugai is committed to creating innovative pharmaceutical products that may satisfy unmet medical needs.
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