Roche Products India Pvt Ltd, a division of Swiss-drugs and biotech giant Roche Group said on Wednesday (17th April) that its Hemophilia A drug emicizumab has been approved in India. It is indicated as a prophylactic (preventive) treatment to prevent or reduce the frequency of bleeding episodes in patients with for Hemophilia A with factor VIII inhibitors. Branded as Hemlibra, this drug is the first weekly subcutaneous (under the skin) prophylaxis injection shown to prevent or reduce the frequency of bleeding episodes and improve the quality of life. It is designed to bring together factor IXa and factor X proteins required to activate the natural coagulation cascade and restore the blood clotting process for people with Hemophilia A.
All current prophylactic treatment options for people with Hemophilia A with factor VIII inhibitors require intravenous infusions several times a week. Even then, some people may experience joint bleeds that can lead to long-term damage.
Roche said in a press statement that the approval of emicizumab is an important advancement for the entire Hemophilia A community. It is a first-in-class of treatments for people with severe Hemophilia A, with inhibitors in nearly 20 years. The clinical evidence of Hemlibra is supported by a comprehensive and extensive development program in Hemophilia A across all ages.
“The introduction of emicizumab is a significant milestone in the treatment of Hemophilia A in India and reaffirms our commitment to bring Roche’s groundbreaking medicines to patients in India as early as possible,” said Lara Bezerra, Chief Purpose Officer (MD), Roche Pharma India.
“This break-through medicine represents a completely new way to manage Hemophilia A and redefines the standard of care. With this new therapy, patients now have a stronger chance of leading a healthy and active life,” she claimed.
According to World Federation of Hemophilia, India records for the highest number of Hemophilia patients in the world. With the current birth rate in India being 32/1000, 1,300 new patients with Hemophilia are born each year. As of 2018 estimates, there are about 50,000 patients suffering from Hemophilia, of which 20,000 people have been identified and there are still about 30,000 unidentified people with Hemophilia in India. Lack of disease awareness and inadequate infrastructure result in high rates of under-diagnosis and sub-optimal treatment, both of which strongly influence not only the quality of life but also the lifespan of people with Hemophilia.
Hemlibra is already approved by multiple regulatory authorities across the world and is now also approved and available in India. In the HAVEN 1 pivotal Phase III clinical study, which was conducted to evaluate the efficacy, safety, and pharmacokinetics of prophylactic emicizumab versus no prophylaxis in Hemophilia A participants with inhibitors, 62.9% of patients had zero bleeds with Hemlibra prophylaxis. In the HAVEN 2 study, 87% of pediatric patients had zero treated bleeds, Roche said.
Hemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Hemophilia A affects around 320,000 people worldwide, approximately 50-60% of whom have a severe form of the disorder. People with Hemophilia A either lack or do not have enough of a clotting protein called factor VIII. In a healthy person, when a bleed occurs, factor VIII brings together the clotting factors IXa and X. It is a critical step in the formation of a blood clot to help stop bleeding. Depending on the severity of their disorder, people with Hemophilia A can bleed frequently, especially into their joints or muscles. These bleeds can present a significant health concern as they often cause pain and can lead to chronic swelling, deformity, reduced mobility, and long-term joint damage. A serious complication of treatment is the development of inhibitors to factor VIII replacement therapies. Inhibitors are antibodies developed by the body’s immune system that bind to and block the efficacy of replacement factor VIII, making it difficult, if not impossible to obtain a level of factor VIII sufficient to control bleeding.
As an active player in the area of haematology, Roche has been developing medicines that have significantly contributed to innovative treatments for blood related diseases in the world for more than 20 years. Its portfolio currently includes rituximab, obinutuzumab) and an anti-CD79b antibody drug conjugate polatuzumab vedotin. Roche’s focus on developing novel molecules in Hematology expands beyond malignancy, with the development of emicizumab, which is a bispecific monoclonal antibody.