FDA Approves Alhemo: A Breakthrough Once-Daily Treatment for Hemophilia A and B with Inhibitors
FDA Approval:
The U.S. Food and Drug Administration (FDA) has approved Alhemo (concizumab-mtci) as a once-daily subcutaneous prophylactic treatment for adults and children aged 12 years and older with hemophilia A or B with inhibitors245.
Mechanism of Action:
Alhemo works by blocking tissue factor pathway inhibitor (TFPI), a protein that prevents blood clotting, thereby increasing the production of thrombin, which is essential for blood clotting234.
Clinical Trial Results:
The pivotal Phase 3 explorer7 study demonstrated an 86% reduction in treated spontaneous and traumatic bleeding episodes in patients on Alhemo prophylaxis compared to no prophylaxis25.
Global Approvals:
Alhemo is also approved in Australia, Japan, Switzerland, and the EU, with specific indications varying by country235.
Patient Benefits:
Alhemo offers a more convenient and less invasive treatment option compared to traditional intravenous infusions, addressing significant unmet needs in managing hemophilia A and B with inhibitors245.
Breakthrough Therapy Designation:
The FDA granted Breakthrough Therapy designation to Alhemo based on results from Phase 2 clinical trials, highlighting its potential to significantly improve the quality of life for individuals living with hemophilia A and B with inhibitors24.
Sources:
2. https://www.novonordisk-us.com/media/news-archive/news-details.html?id=915084
3. https://www.globenewswire.com/news-release/2024/10/18/2965407/0/en/Novo-Nordisk-A-S-Alhemo-recommended-for-European-approval-as-first-once-daily-subcutaneous-prophylactic-treatment-for-people-living-with-haemophilia-A-or-B-with-inhibitors.html
4. https://www.managedhealthcareexecutive.com/view/alhemo-once-daily-treatment-for-hemophilia-a-b-with-inhibitors-fda-approved
5. https://www.ajmc.com/view/first-subcutaneous-prophylaxis-for-hemophilia-a-and-b-with-inhibitors-approved-by-fda