On December 20, 2024, FDA approved Alhemo (concizumab-mtci) injection to reduce the hemophilia related bleeding frequency in patients of 12 years and older. This is a first of its kind subcutaneous injection treatment for patients suffering from hemophilia A with factor 8 inhibitors or hemophilia B with factor 11 inhibitors
Who is the treatment target?
“I look at hemophilia and see that it’s a part of me. I’m not going to work my way around it. I’m going to work with it. I’m not going to let it define me.” This is the story of Alden Dunlap, a 23-year-old man suffering from severe hemophilia for most of his life.
Hemophilia is a rare, inherited bleeding disorder that primarily affects males. It is caused by a deficiency in specific clotting factors in the blood, leading to excessive bleeding or spontaneous bleeding episodes. In the United States, it is estimated that as many as 33,000 males are living with hemophilia. Hemophilia A is the most common type, affecting about 3-4 times more people than hemophilia B. The severity of hemophilia varies, with severe cases requiring frequent medical intervention. Early diagnosis and treatment are crucial to prevent complications such as joint damage and internal bleeding.
How does Vyloy work?
Inhibitor development complicates hemophilia treatment, affecting about 30% of individuals with severe hemophilia A and 5-10% of those with severe hemophilia B.0020Alhemo® works by targeting tissue factor pathway inhibitor (TFPI), a protein that regulates blood clotting. A person with hemophilia A with inhibitors has a bleeding episode when the body’s natural clotting process is disrupted. TFPI can further inhibit clotting, making it difficult for the blood to form a clot and stop bleeding.
Alhemo® binds to TFPI, preventing it from interfering with the clotting process. This allows the remaining clotting factors in the body to work more effectively, promoting clot formation and helping to control bleeding episodes.
Clinical Studies, Efficacy and and Side Effects
Data from Explorer7, a phase 3 trial was used to investigate the efficacy and safety of concizumab in individuals with hemophilia A or B with inhibitors. Participants were randomized to receive either immediate concizumab prophylaxis or on-demand treatment followed by concizumab prophylaxis. Compared to those without prophylaxis, patients on Alhemo® experienced an 86% decrease in the Annualized Bleeding Rate (ABR), with a mean ABR of 1.7 versus 11.8, respectively.
Notably, 64% of patients on Alhemo® prophylaxis experienced zero treated bleeds within the first 24 weeks of treatment, highlighting its potential to significantly improve bleeding control in this population. While generally well-tolerated, the most common adverse reactions included injection site reactions and urticaria.
Market and upcoming competitors
The current treatment for hemophilia varies from concentrates of clotting factor to non-factor VIII products like Hemlibra, which mimic factor VIII function, desmopressin (DDAVP) to stimulate the body’s own factor VIII production, and Amicar to stabilize existing clots. Alhemo joins the fight as an alternative which might increase the option and reduce the treatment cost, reducing the burdens from suffering patients.
Further reads
https://www.novonordisk-us.com/media/news-archive/news-details.html?id=915084
https://www.cdc.gov/hemophilia/stories/personal-story-alden.html
https://clinicaltrials.gov/study/NCT04083781
https://www.cdc.gov/hemophilia/treatment/index.html
Avraneel Paul, Ph.D.
Disclaimer
The editors take care to share authentic information. In case of any discrepancies please write to newsletter@medness.org
The sponsors do not have any influence on the nature or kind of the news/analysis reported in MedNess. The views and opinions expressed in this article are those of the authors and do not necessarily reflect the official policy or position of MedNess. Examples of analysis performed within this article are only examples. They should not be utilized in real-world analytic products as they are based only on very limited and dated open-source information. Assumptions made within the analysis are not reflective of the position of anyone volunteering or working for MedNess. This blog is strictly for news and information. It does not provide medical advice, diagnosis or treatment nor investment suggestions. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
MedNess is a part of STEMPeers® which is a 501(c)(3) organization registered in PA as PhD Career Support Group. The organization helps create a growing network of STEM scientists that is involved in peer-to-peer mentoring and support.
