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Malaysian Reserve
31-07-2025
- Health
- Malaysian Reserve
FDA approves Alhemo® as once-daily prophylactic treatment to prevent or reduce the frequency of bleeding episodes for adults and children 12 years of age and older with hemophilia A or B (HA/HB) witho
FDA approval is based on phase 3 trial data (explorer8), which established the safety and efficacy of Alhemo® (concizumab-mtci) injection in people 12 years and older with hemophilia A or B (HA/HB) without inhibitors1 Results showed a 79% reduction in annualized bleeding rate (ABR) in patients with HB without inhibitors and an 86% reduction in patients with HA without inhibitors, respectively, using Alhemo® prophylaxis compared to no prophylaxis2 Alhemo® was the first subcutaneous injectable pen of its kind for patients living with HA and HB with inhibitors and is now approved for those living without inhibitors1 PLAINSBORO, N.J., July 31, 2025 /PRNewswire/ — Novo Nordisk announced today that the US Food and Drug Administration (FDA) approved Alhemo® (concizumab-mtci) injection as a once-daily prophylaxis 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 (HA/HB) without inhibitors, expanding on the December 2024 approval for HA/HB with inhibitors.1,2,3 Currently, many treatments for HA/HB without inhibitors are administered via intravenous infusions.2 With this approval, Alhemo® now offers a subcutaneous injection treatment option for this population.1 'The FDA approval of an expanded indication for Alhemo® marks a meaningful step forward for people with hemophilia A or B without inhibitors who are looking for a new prophylaxis treatment option,' said Anna Windle, PhD, Senior Vice President, Clinical Development, Medical & Regulatory Affairs, Novo Nordisk. 'By building on the initial indication for Alhemo® for those with hemophilia with inhibitors—an especially significant development in hemophilia B where challenges still exist despite advanced treatment options—Novo Nordisk continues its 35+ year legacy in rare bleeding disorders to continue to help address the unmet needs of this community.' Alhemo® is designed to block a protein called tissue factor pathway inhibitor (TFPI), which stops blood from clotting. By inhibiting TFPI, Alhemo® improves the production of thrombin, which helps to clot the blood and prevent bleeding, when clotting factors VIII and IX are missing or deficient regardless of inhibitor status.4,5 'For people living with hemophilia, it is important to continually monitor and discuss bleed control with their healthcare professional,' said Allison P. Wheeler, MD, Scientific Director, Washington Center for Bleeding Disorders, Seattle, WA. 'With today's approval of Alhemo® for hemophilia A or B without inhibitors, more people living with these rare blood disorders now have a daily prophylaxis option that may help decrease their bleeding rates.' The primary objective of the pivotal phase 3 explorer8 trial was to compare the number of treated spontaneous and traumatic bleeding episodes, as measured by the ABR, in patients aged 12 years and older with HA/HB without inhibitors, receiving Alhemo® prophylaxis versus no prophylaxis (on demand Factor VIII/Factor IX treatment).2 The study showed a statistically significant reduction in ABR of 79% for HB patients (ABR ratio: 0.21; 95% CI: 0.10–0.45; p<0.0001) and 86% for HA patients (ABR ratio: 0.14; 95% CI: 0.07–0.29; p<0.0001). Additionally, notable reductions in the average and median ABRs further reinforced the efficacy of Alhemo® prophylaxis in reducing bleeds across both patient groups, with an average ABR of 3.1 (1.9-5.0) and a median ABR of 1.6 (0.0-4.8) for HB patients, compared to 14.8 (8.1-26.9) and 14.9 (3.3-22.1), respectively, and an average ABR of 2.7 (1.6-4.6) and median ABR of 2.9 (0.0-5.2) for HA patients, compared to 19.3 (11.3-33.0) and 19.6 (17.3-30.4), respectively. In the study, the most common adverse reactions reported in ≥5% of patients randomized to receive Alhemo® were injection site reactions (7%) and headache (7%).1 Alhemo® is provided in prefilled, premixed pens for subcutaneous injection (60 mg/1.5 mL, 150 mg/1.5 mL, or 300 mg/3 mL) via thin 32 gauge, 4 mm needles, which are provided separately, offering an additional treatment choice for patients with hemophilia with inhibitors – and now without inhibitors – to further customize their care. About the explorer8 studyExplorer8 is a phase 3 clinical trial that evaluated the efficacy and safety of Alhemo® for adults and pediatric patients 12 years of age and older living with hemophilia A or B (HA/HB) without inhibitors.2 In explorer8, among the 156 males with HA/HB included in the trial, 63 were randomly assigned in a 1:2 ratio to receive no prophylaxis (arm 1, n=21), or Alhemo® prophylaxis (arm 2, n=42).2,6 The median duration of treatment was 24.1 weeks in arm 1 (no prophylaxis) and 32.1 weeks in arm 2 (Alhemo® prophylaxis).The initial loading dose of Alhemo® was 1 mg per kilogram of body weight, followed by 0.2 mg per kilogram daily, and potentially individualized on the basis of concizumab-mtci plasma concentration as measured at week 4.1,2 Efficacy was evaluated separately in HA patients without inhibitors and in HB patients without inhibitors when all patients in arms 1 and 2 had completed the main part of the trial (at least 24 or at least 32 weeks, respectively), based on the number of treated bleeding episodes comparing Alhemo® prophylaxis and no prophylaxis.1 About Alhemo® (concizumab-mtci) Alhemo® is a tissue factor pathway inhibitor (TFPI) antagonist. By inhibiting the TFPI, Alhemo® enhances factor Xa (FXa) production during the initiation phase of coagulation, leading to improved thrombin generation and clot formation in patients with hemophilia A or B (HA/HB) with or without inhibitors. The effect of Alhemo® is not influenced by the presence of inhibitory antibodies to FVIII or FIX and Alhemo® does not induce or enhance the development of direct inhibitors to FVIII or FIX. Alhemo® is approved as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12 years of age and older with HA/HB with or without inhibitors in the US.1 What is Alhemo®?Alhemo® (concizumab-mtci) injection 60 mg, 150 mg, or 300 mg is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children 12 years of age and older with hemophilia A or B with or without factor VIII or IX inhibitors. It is not known if Alhemo® is safe and effective in people receiving ongoing immune tolerance induction (ITI) or in children younger than 12 years of age Important Safety Information What is the most important information I should know about Alhemo®? It is important to follow the daily dosing schedule of Alhemo® to stay protected against bleeding. This is especially important during the first 4 weeks of treatment to make sure a correct maintenance dose is established. Use Alhemo® exactly as prescribed by your healthcare provider (HCP). Do not stop using Alhemo® without talking to your HCP. If you miss doses or stop using Alhemo®, you may no longer be protected against bleeding Your HCP may prescribe factor VIII, factor IX, or bypassing agents during treatment with Alhemo®. Carefully follow your HCP's instructions regarding when to use on-demand factor VIII, factor IX, or bypassing agents and the recommended dose and schedule for breakthrough bleeds Do not use Alhemo® if you are allergic to concizumab-mtci or any of the ingredients in Alhemo®. Before using Alhemo®, tell your HCP about all of your medical conditions, including if you: Have a planned surgery. Talk to your HCP about when to stop using Alhemo® and when to start it again if you have a planned surgery Are pregnant, breastfeeding, or plan to become pregnant or breastfeed. It is not known if Alhemo® may harm your unborn baby or if Alhemo® passes into your breast milk Your HCP may do a pregnancy test before you start treatment with Alhemo® Females who are able to become pregnant, talk to your HCP about using effective birth control (contraception) methods during treatment with Alhemo® and for 7 weeks after ending treatment Your HCP may do a pregnancy test before you start treatment with Alhemo® Females who are able to become pregnant, talk to your HCP about using effective birth control (contraception) methods during treatment with Alhemo® and for 7 weeks after ending treatment Tell your HCP about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and supplements. What are the possible side effects of Alhemo®?Alhemo® may cause serious side effects, including: Blood clots (thromboembolic events). Alhemo® may cause blood clots to form in blood vessels, such as in your arms, legs, heart, lung, brain, eyes, kidneys, or stomach. You may be at risk for getting blood clots if you use high or frequent doses of factor products or bypassing agents to treat breakthrough bleeds, or if you have certain conditions. Get medical help right away if you have any signs and symptoms of blood clots, including: swelling, warmth, pain, or redness of the skin; headache; trouble speaking or moving; eye pain or swelling; sudden pain in your stomach or lower back area; feeling short of breath or severe chest pain; confusion; numbness in your face; and problems with your vision Allergic reactions. Alhemo® can cause allergic reactions, including redness of the skin, rash, hives, itching, and stomach-area (abdominal) pain. Stop using Alhemo® and get emergency medical help right away if you develop any signs or symptoms of a severe allergic reaction, including: itching on large areas of skin; trouble swallowing; wheezing; pale and cold skin; dizziness due to low blood pressure; redness or swelling of lips, tongue, face, or hands; shortness of breath; tightness of the chest; and fast heartbeat The most common side effects of Alhemo® include: bruising, redness, bleeding, itching, rash or lump at the injection site, headache, and hives. These are not all the possible side effects of Alhemo®. Call your doctor for medical advice about side effects. Please click HERE for Alhemo® Prescribing Information and Medication Guide About hemophiliaHemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding.7 It is estimated to affect approximately ~800,000 people worldwide.8 There are different types of hemophilia, which are characterized by the type of clotting factor protein that is defective or missing. Hemophilia A is caused by a missing or defective clotting Factor VIII (FVIII), and hemophilia B is caused by a missing or defective clotting Factor IX (FIX).7 Hemophilia is often treated by replacing the missing clotting factor via intravenous infusions, also known as replacement therapy.8 About Novo Nordisk Novo Nordisk is a leading global healthcare company that's been making innovative medicines to help people with diabetes lead longer, healthier lives for more than 100 years. This heritage has given us experience and capabilities that also enable us to drive change to help people defeat other serious chronic diseases such as obesity, rare blood, and endocrine disorders. We remain steadfast in our conviction that the formula for lasting success is to stay focused, think long-term, and do business in a financially, socially, and environmentally responsible way. With a US presence spanning 40 years, Novo Nordisk US is headquartered in New Jersey and employs over 10,000 people throughout the country across 12 manufacturing, R&D, and corporate locations in eight states plus Washington DC. For more information, visit Facebook, Instagram, and X. References Alhemo® (concizumab-mtci) injection, for subcutaneous use [package insert]. Plainsboro, NJ: Novo Nordisk Inc. Chowdary P., et al. Concizumab prophylaxis in people with haemophilia A or haemophilia B without inhibitors (explorer8): a prospective, multicentre, open-label, randomised, phase 3a trial. Lancet Hematology. 2024;11:e891-904. Novo Nordisk. FDA approves drug to prevent or reduce the frequency of bleeding episodes for patients with hemophilia A with inhibitors or hemophilia B with inhibitors. Accessed May 2025. Available at Shapiro AD. Concizumab: a novel anti-TFPI therapeutic for hemophilia. Blood Adv. 2021;5(1):279. Matsushita T, Shapiro A, Abraham A, et al. Phase 3 trial of concizumab in hemophilia with inhibitors. N Engl J Med. 2023; 389(9): 783-794. Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors (explorer8). Accessed May 2025. Available at MedlinePlus. Hemophilia. Accessed May 2025. Available at World Federation of Hemophilia. Annual Global Survey 2023. Accessed July 2025. Further information Liz Skrbkova (US)+1 609 917 0632 (Mobile)USMediaRelations@ Ambre James-Brown (Global)+45 3079 9289Globalmedia@ Alhemo® is a registered trademark of Novo Nordisk Health Care AG. Novo Nordisk is a registered trademark of Novo Nordisk A/S.© 2025 Novo Nordisk All rights reserved. US25AHM00187 July 2025
Business Upturn
22-06-2025
- Health
- Business Upturn
Novo Nordisk A/S: Mim8 prophylaxis treatment shown to be well-tolerated when switching from emicizumab in people with haemophilia A in new phase 3 data presented at the ISTH 2025 Congress
By GlobeNewswire Published on June 23, 2025, 00:35 IST New FRONTIER5 data show that a direct switch to investigational Mim8 (denecimig) prophylaxis treatment from emicizumab, without the need for a washout period, was well-tolerated with no safety concerns in adults and adolescents with haemophilia A, with or without inhibitors 1 . . Switching to Mim8 led to a sustained increase in thrombin generation into the normal range, but without causing thrombin levels that might pose a thrombotic risk 1 . . FRONTIER5 Patient-Reported Outcomes (PROs) assessment found the Mim8 pen-injector easy to use, with strong user preference over their emicizumab injection system 2 . . These results add to the overall safety profile of Mim8 based on the FRONTIER clinical trial programme3. Bagsværd, Denmark, 22 June 2025 – Novo Nordisk today presented results from the phase 3b FRONTIER5 trial showing that a direct switch to investigational Mim8 (denecimig) prophylaxis from emicizumab treatment, without a washout period or Mim8 loading dose, was well-tolerated with no safety concerns in adults and adolescents living with haemophilia A, with or without inhibitors1. Additionally, a FRONTIER5 Patient-Reported Outcomes (PROs) assessment found the Mim8 pen-injector easy to use, with an overall strong user preference for the pen-injector compared to the previous emicizumab injection system2,3. The results were presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C. In the study, the first Mim8 maintenance dose was administered on the next planned emicizumab dosing day. Patients were given the option of switching to once-monthly, once every two weeks or once-weekly dosing frequencies of Mim8, regardless of their prior dosing frequency1,3. Steady-state Mim8 concentration was achieved by Week 16, and emicizumab elimination was completed by Week 261. Switching to Mim8 led to a sustained increase in thrombin peak levels without an exaggerated thrombin response1. 'Continuous prophylactic coverage is critical to avoiding breakthrough bleeds in people living with haemophilia; with new non-factor therapeutic options, many people could have hesitations about switching treatment options. These data demonstrate that switching to Mim8 from emicizumab can be done without requiring a washout period,' said Allison P. Wheeler, MD, Washington Center for Bleeding Disorders, Seattle, WA. 'This is critical in ensuring that individuals maintain continuous protection against bleeding events as we seek to help address the ongoing needs of people living with this complex disease.' The open-label phase 3b FRONTIER5 study consisted of 61 adults and adolescents, aged 12 years and older, with haemophilia A. Mim8 was well-tolerated with no safety concerns. No thromboembolic events, hypersensitivity reactions, or treatment-emergent adverse events (TEAEs) leading to discontinuation were observed, and there was no clinical evidence of neutralising anti-Mim8 antibodies1. The PROs data from FRONTIER5 indicated a strong overall preference for the Mim8 pen-injector, with 97% (n=57/59) of patients reporting a 'very strong' or 'fairly strong' preference in comparison to their previous emicizumab injection system2. Of the participants who completed the Haemophilia Device Handling and Preference Assessment (HDHPA) questionnaire at week 26, 98% (n=58/59) found the Mim8 pen-injector 'very easy' or 'easy' to use, and 95% (n=56/59) found it 'much easier' or 'easier' compared with their previous administration method. All participants (100%) were 'extremely confident' or 'very confident' in using the pen-injector correctly, and most participants (83%; n=49/59) found it 'very easy' to inject the dose2. 'The FRONTIER5 safety and patient-reported outcomes data support Mim8 as a potential future treatment option for people living with haemophilia A and demonstrate our continued commitment to developing innovative treatment options for this community', said Stephanie Seremetis, chief medical officer and CVP for Haemophilia at Novo Nordisk. 'These results give valuable insights into haemophilia A management, highlight the feasibility of directly switching to Mim8 from emicizumab, and reveal a strong patient preference for the Mim8 pen-injector device.' Novo Nordisk expects to submit Mim8 for regulatory review during 2025. Data from the ongoing phase 3 FRONTIER programme will be disclosed at upcoming congresses and in publications in 2025 and 2026. About haemophilia Haemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding4. It is estimated to affect approximately 1,125,000 people worldwide5. There are different types of haemophilia, which are characterised by the type of clotting factor protein that is defective or missing4. Haemophilia A is caused by a missing or defective clotting Factor VIII (FVIII), and haemophilia B is caused by a missing or defective clotting Factor IX4. Inhibitors are an immune system response to the clotting factors in replacement therapy. Currently, it is estimated that up to 30% of people living with severe haemophilia A develop inhibitors6 that can cause replacement therapies to stop working. About Mim8 Mim8 is an investigational FVIIIa mimetic bispecific antibody optimised with the aim to deliver improved potency and sustained efficacy across flexible dosing intervals up to once-monthly prophylaxis for people living with haemophilia A, with or without inhibitors7-10. Administered under the skin, Mim8 bridges Factor IXa and Factor X. This action replaces FVIII function, which helps restore the body's thrombin generation capacity into the normal range, helping blood to clot7,11. The use of Mim8 in people living with haemophilia A is investigational and not approved by regulatory authorities or available anywhere in the world. About the FRONTIER5 trial FRONTIER5 is a single-arm, open-label, 26-week, phase 3b trial evaluating the safety of switching from previous emicizumab prophylaxis treatment directly to Mim8 prophylaxis treatment using the Mim8 pen-injector in adults and adolescents with haemophilia A, with or without inhibitors3. The FRONTIER clinical programme investigates Mim8 as a prophylaxis treatment for people with haemophilia A, with or without inhibitors. This programme includes FRONTIER1, FRONTIER2, FRONTIER3, FRONTIER4 and FRONTIER53,12-15. About Novo Nordisk Novo Nordisk is a leading global healthcare company founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 77,400 people in 80 countries and markets its products in around 170 countries. For more information, visit , Facebook , Instagram , X , LinkedIn and YouTube . Contacts for further information _______________________ References Oldenberg J, Benson G, Chowdaryet P, et al. FRONTIER5 direct switch study: Safety of initiating Mim8 prophylaxis without washout of emicizumab. Oral presentation presented at the Congress of the International Society on Thrombosis and Haemostasis 2025; June 21-25 2025; Walter E. Washington Convention Center, Washington D.C., US. Session code 13686. Mahlangu J, Ahuja S, Cockrell E, et al. FRONTIER5 device handling and patient-reported outcomes. Oral presentation presented at the Congress of the International Society on Thrombosis and Haemostasis 2025; June 21–25 2025; Walter E. Washington Convention Center, Washington D.C., US. Session code 13786. A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER5). Available at: Last accessed: June 2025. MedlinePlus. Hemophilia. Available at: Last accessed: June 2025. Iorio A, Stonebraker JS, Chambost H, et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries. Ann Intern Med. 2019;171:540–546. doi: 10.7326/M19-1208. Kim JY, You CW. The prevalence and risk factors of inhibitor development of FVIII in previously treated patients with hemophilia A. Blood Res. 2019;54:204-209. doi: 10.5045/br.2019.54.3.204. Ostergaard H, Lund J, Greisen PJ, et al. A factor VIIIa-mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia A mice. Blood. 2021;138:1258-1268. doi: 10.1182/blood.2020010331. Mancuso EM, et al. Efficacy and safety of Mim8 prophylaxis in adults and adolescents with hemophilia A with or without inhibitors: Phase 3, open-label, randomized, controlled FRONTIER2 study. Abstract presented at the International Society on Thrombosis and Haemostasis (ISTH) 2024 Congress. Kenet G, et al. Patient- and caregiver-reported outcomes with subcutaneous Mim8 prophylaxis in paediatric patients with haemophilia A with or without factor VIII inhibitors: phase 3 FRONTIER3 study. Abstract presented at the European Association for Haemophilia and Allied Disorders (EAHAD) 2025 Annual Congress. Session 6. Chowdary P, Banchev AM, Kavakli K, et al. Safety and Efficacy of Mim8 Prophylaxis Administered Once Every Two Weeks for Patients with Hemophilia A with or without Inhibitors: Interim Analysis of the FRONTIER4 Open-Label Extension Study. Abstract presented at the American Society of Hematology (ASH) 2024 Annual Congress. Session: 322. U.S. National Library of Medicine. F8 gene. MedlinePlus Genetics. Available at Last accessed: June 2025. A Research Study Investigating Mim8 in People With Haemophilia A (FRONTIER1). Available at: Last accessed: June 2025. A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors. Available at: Last accessed: June 2025. A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors. Available at: Last accessed: June 2025. A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A (FRONTIER4). Available at: Last accessed: June 2025. Attachment Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. Ahmedabad Plane Crash GlobeNewswire provides press release distribution services globally, with substantial operations in North America and Europe.
