Marstacimab-hncq

Novo Nordisk finally won US approval for its hemophilia drug Alhemo following the product’s rejection by the FDA last year. The once-daily shot may now be used to reduce the frequency of bleeding episodes or prevent them entirely in adults and children aged 12 and up with hemophilia A or B with inhibitors. The company didn’t provide a US price for the drug. Friday’s approval gives the Danish pharma some good news to end the year — though not enough to erase the damage done by its underwhelming CagriSema readout , which emerged earlier on Friday. The shares gained about 5% in early trading Monday, after losing almost a fifth of their value Friday following the CagriSema data release. Alhemo, previously known as concizumab, is an anti-tissue factor pathway inhibitor (TFPI). By blocking TFPI, Alhemo raises levels of thrombin, a protein that helps blood clot in the presence of inhibitors. Inhibitors are antibodies patients can develop as part of their immune system; they bind to and neutralize clotting factors such as factor VIII or factor IX. In its pivotal trial, Alhemo cut the frequency of treated spontaneous and traumatic bleeds by 86% compared with patients who had no preventive treatment. Novo’s new drug is given subcutaneously using a pen device, available in three doses. The company claims this is the first subcutaneous treatment of its kind for this population, with “many” of the other treatments being intravenous. Pfizer’s Hympavzi, approved in the US in October , has the same mechanism and is also delivered subcutaneously using a pen injector, though on a weekly rather than daily schedule. However, Hympavzi is only approved for hemophilia A and B patients without inhibitors. According to Novo, an estimated 30% of severe hemophilia A patients and 5-10% of severe hemophilia B patients will develop inhibitors, so theoretically Alhemo could reach a wider population than Pfizer’s product. But Pfizer has plans to fight back: data on Hympavzi in a cohort of patients with inhibitors should arrive in 2025. These results could form the basis of a label expansion that would put Pfizer’s drug on equal regulatory footing with Alhemo but with an obvious convenience advantage. The big beast here is Roche’s weekly shot Hemlibra, which had sales of around $4.6 billion last year. This is approved for hemophilia A patients with or without inhibitors, but cannot treat the B form of the disease. Novo is aiming to take Hemlibra on not with Alhemo but with another asset, Mim8, which passed its Phase 3 test in May. Here, Novo may have the convenience edge, since Mim8 can be given once a month.

In the current global pharmaceutical market, gene therapy drugs occupy a significant portion of the most expensive drug rankings, with five listed in the top ten. Pfizer's recently approved treatment for moderate to severe Hemophilia B, Beqvez, is priced at a staggering $3.5 million, making it one of the highest-priced drugs in history. Hemophilia is a hereditary bleeding disorder primarily divided into Hemophilia A and B, caused by mutations in the genes for clotting factors VIII and IX, respectively. Patients typically suffer from lifelong coagulation dysfunction, leading to joint bleeding and other severe complications. Currently, replacement therapies with clotting factors are the main treatment method, but frequent long-term injections can lead to poor patient compliance and may produce inhibitory antibodies, affecting treatment efficacy. Although new clotting factor drugs, humanized bispecific antibodies, and monoclonal antibodies that inhibit the tissue factor pathway have made advancements in administration methods and duration of action, these methods still cannot cure hemophilia. For hemophilia patients, gene therapy offers a glimmer of hope for a cure. Through gene therapy, clotting factors can be safely delivered into the patient's cells for long-term stable expression, thereby improving bleeding conditions and coagulation function, which is the ultimate goal of hemophilia treatment. Currently, several gene therapy drugs for hemophilia have been approved, including: HYMPAVZI™ (Matizumab): An anti-TFPI antibody suitable for patients with Hemophilia A or B, developed by Pfizer, approved by the European Commission in November 2024. Vonvendi (Veyinzh®): A recombinant von Willebrand factor (rVWF) for adult patients with von Willebrand disease (VWD), developed by Takeda, approved by China's National Medical Products Administration in August 2024. Beqvez: A neutralizing antibody for adult patients with moderate to severe Hemophilia B, developed by Pfizer, priced at $3.5 million. Hemgenix (AMT060): A gene therapy based on adeno-associated virus 5 (AAV5) for patients with Hemophilia B, developed by UniQure, approved by the U.S. FDA in November 2022. Roctavian: Also based on the AAV5 vector, this gene therapy is for patients with Hemophilia A (HA), priced at $2.9 million per treatment, developed by BioMarin, and received conditional marketing authorization from the European Commission in August 2022. As the hemophilia market continues to grow, it’s expected to reach a scale of billions by 2028. Currently, over 40 gene therapies for hemophilia are in research, with multinational companies like Pfizer, Sanofi, Bayer, and Takeda, as well as domestic firms like China National Pharmaceutical Group and Hualan Biologicals, actively conducting clinical studies. Despite the enormous potential of gene therapy in treating hemophilia, the high costs remain a significant barrier to its widespread adoption.