Groundbreaking Drug Launches
Concizumab (Alhemo)
Mechanism of Action: Lipoprotein-associated coagulation inhibitor inhibitors Drug class: Monoclonal antibodies Originator and Owner: Novo Nordisk Indication: Haemophilia A; Haemophilia B
Approval Date: 20 December 2024 Trials: explorer7
Haemophilia A
Haemophilia A is a genetic bleeding disorder caused by a lack of factor VIII, resulting in prolonged and excessive bleeding either spontaneously or following injury. Von Willebrand disease is another bleeding disorder characterized by low levels of clotting protein in the blood. Various medications, such as Advate, Recombinate, Eloctate, and Hemlibra, are available for treating hemophilia A. However, a major challenge in treatment is the development of inhibitors, which prevent factor treatment from being effective in slowing or stopping bleeding episodes.
Haemophilia B
Haemophilia B is a rare genetic bleeding disorder that occurs when a gene responsible for blood clotting undergoes a mutation or change. Without treatment, haemophilia B can be life-threatening. Researchers are exploring gene therapy and gene replacement therapy as potential new treatments for haemophilia B and other types of haemophilia.
Prevalence Haemophilia A
According to the Centers for Disease Control and Prevention (October 2023), haemophilia A occurs in one out of 5000 males and accounts for 80% of cases. More than 400,000 males worldwide are affected by haemophilia A, with a significant number going undiagnosed in developing countries.
Prevalence Haemophilia B
Experts estimate that approximately 4 in 100,000 men and individuals designated male at birth (DMAB) in the United States have haemophilia B. While women and individuals designated female at birth (DFAB) can also have haemophilia B, it is difficult to determine how many are affected. This is because their symptoms, such as heavy menstrual bleeding or excessive postpartum bleeding, are often not recognized as being related to haemophilia.
Challenges in the treatment of haemophilia with current medications
Inhibitor Development • Neutralizing Antibodies: Patients may develop neutralizing antibodies, also known as inhibitors, against the infused clotting factor. This can complicate the treatment of bleeding episodes and raise the overall cost of care. •Treatment Options: Bypassing agents (BPAs) are commonly used to treat patients with inhibitors.
Cost • Coagulation Factor Concentrates: The high cost of coagulation factor concentrates can be a barrier to treatment, especially in developing countries. • Inhibitors: The presence of inhibitors further increases the cost of care.
Why is Concizumab poised to make an impact in 2025?
The US FDA approved Concizumab (Alhemo) to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B with inhibitors. Novo Nordisk has received support from European regulators for the approval of its anti-TFPI monoclonal antibody, Alhemo (concizumab), as a prophylactic treatment for haemophilia. Alhemo would be the first once-daily subcutaneous prophylactic treatment for individuals with haemophilia A or B who have inhibitors.
Although the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has cleared a significant hurdle with its recommendation, the launch of Alhemo is likely to present several challenges for Novo Nordisk. These include competing with established, effective treatments like Roche’s Hemlibra (emicizumab) and Sanofi’s Altuviiio (efanesoctocog alfa), as well as facing anticipated competition from Pfizer’s once-weekly anti-TFPI antibody, marstacimab.
“First once-daily subcutaneous treatment for haemophilia A or B with inhibitors”
Regulatory milestones
Registrational Trials
The US FDA has approved Alhemo (concizumab injection as a once-daily prophylaxis for preventing bleeding in adults and pediatric patients 12 and older with hemophilia A or B with inhibitors, and the EMA's CHMP has recommended its approval as the first once-daily subcutaneous treatment for the same group, based on data of explorer 7 trial.
explorer 7:
A phase 3 clinical trial aimed to evaluate the safety and efficacy of concizumab, a monoclonal antibody, as a once-daily subcutaneous prophylactic treatment for individuals aged 12 years and older with hemophilia A or B who have developed inhibitors (NCT04083781).
“Concizumab (SC) reduced annualized bleeding rates in haemophilia A or B patients with inhibitors vs no prophylaxis”