Delving into the Latest Updates on Emicizumab with Synapse

Overview

Basic Info

Drug Type

Bispecific antibody

Synonyms

Anti-factor IXa anti-factor X humanized bispecific antibody, Emicizumab (Genetical Recombination), Emicizumab (genetical recombination) (JAN)

+ [14]

Target

F10 x factor IXa

Action

inhibitors

Mechanism

F10 inhibitors(Coagulation factor X inhibitors), factor IXa inhibitors(Factor IXa inhibitors)

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication

Coagulation Protein DisordersHemophilia AFactor 8 Deficiency, Acquired+ [1]

Inactive Indication-

Originator Organization

Chugai Pharmaceutical Co., Ltd.

Active Organization

Chugai Pharmaceutical Co., Ltd.

Roche China Holding Ltd.Hoffmann-La Roche, Inc.

+ [7]

Inactive Organization-

Drug Highest PhaseApproved

First Approval Date

United States (16 Nov 2017),

Hemophilia A

RegulationOrphan Drug (United States), Priority Review (China), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Breakthrough Therapy (United States), Orphan Drug (Japan)

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Structure/Sequence

Sequence Code 5386H

Source: *****

Sequence Code 5425H

Source: *****

Sequence Code 5467L

Source: *****

We have developed a questionnaire to elucidate the dosing, frequency and indication for the use of emicizumab in patients with Hemophilia A (mild, moderate or severe) ages 0-3 years. We are also collecting data on any pre-, peri and post surgical practices while on emicizumab. More importantly, we are asking if pediatricians are planning to introduce factor 8 to children who are already on emicizumab for primary prophylaxis as well as how and when they are planning to do so. We hope that this data will help inform understanding of current use of emicizumab in infants and young children as a form of primary prophylaxis, especially when venous access has historically been a limiting factor.

Start Date01 Aug 2024

Sponsor / Collaborator

Montefiore Medical Center

[+1]

100 Clinical Results associated with Emicizumab

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100 Translational Medicine associated with Emicizumab

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100 Patents (Medical) associated with Emicizumab

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656

Literatures (Medical) associated with Emicizumab

31 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

Systematic review of cost-effectiveness modelling studies for haemophilia

Review

Author: Ammann, Daniel ; Meier, Niklaus ; Schwenkglenks, Matthias ; Probst, Jano ; Pletscher, Mark

AIMS:

Haemophilia is a rare genetic disease that hinders blood clotting. We aimed to review model-based cost-effectiveness analyses (CEAs) of haemophilia treatments, describe the sources of clinical evidence used by these CEAs, summarize the reported cost-effectiveness of different treatment strategies, and assess the quality and risk of bias.

METHODS:

We conducted a systematic literature review of model-based CEAs of haemophilia treatments by searching databases, the Tufts Medical Center CEA registry, and grey literature. We summarized and qualitatively synthesized the approaches and results of the included CEAs, without a meta-analysis due the diversity of the studies.

RESULTS:

32 eligible studies were performed in 12 countries and reported 53 pairwise comparisons. Most studies analysed patients with haemophilia A rather than haemophilia B. Comparisons of prophylactic versus on-demand treatment indicated that prophylaxis may not be cost-effective, but there was no clear consensus. Emicizumab was generally cost-effective compared with clotting factor treatments and was always dominant for patients with inhibitors. Immune tolerance induction following a Malmö protocol was found to be cost-effective compared to bypassing agents, while there was no consensus for the other protocols. Gene therapies as well as treatment with extended half-life coagulation factors were always cost-effective over their comparators. Studies were highly heterogenous regarding their time horizons, model structures, the inclusion of bleeding-related mortality and quality-of-life impacts. This heterogeneity limited the comparability of the studies. 19 of the 32 included studies received industry funding, which may have biased their results.

LIMITATIONS:

It was not possible to perform a quantitative synthesis of the results due to the heterogeneity of the underlying studies.

CONCLUSION:

Differences in results between previous CEAs may have been driven by heterogeneity in modelling approaches, clinical input data, and potential funding biases. A more consistent evidence base and modelling approach would enhance the comparability between CEAs.

01 Mar 2025·JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY

A 5-Year Single-center Experience on the Use of Emicizumab Prophylaxis in Children With Severe Hemophilia A With and Without Factor VIII Inhibitors

Article

Author: Motwani, Jayashree ; Ahmed Hasan, Esraa Mohamed

Objective::

Emicizumab promotes efficacious hemostasis in persons with hemophilia A persons with hemophilia A with and without inhibitors. Primary analyses of real-world data and clinical trials have shown emicizumab efficacy and safety; however, long-term data are limited.

Methods::

This retrospective study was conducted to assess real-world long-term outcomes of pediatric patients on emicizumab in our hemophilia center between the period of February 2018 and September 2023. Relevant demographic and clinical data were gathered.

Results::

Seventy-eight patients were enrolled. Previously untreated patients and minimally treated patients accounted for 14.1% and 10.3% of our recruited patients, respectively. One of 5 patients with active inhibitors experienced a single recombinant activated factor VII-treated bleeding episode while on emicizumab prophylaxis. Twenty-eight (28/78) patients underwent surgical/dental procedures without bleeding complications except for 2 patients (7.1%). Thirty-three patients experienced 62 factor-treated bleeding episodes with only 11 imaging-confirmed joint/muscle bleeding episodes among 8 patients. No major safety concerns were reported in the study and emicizumab was discontinued in 5 patients (2 for antidrug antibody development, 2 for joint bleeding, and 1 for non-compliance).

Conclusion::

Emicizumab prophylaxis was well tolerated with no new safety concerns, and 45 patients (57.7%) of our cohort exhibited zero-treated bleeds, which was comparable to other published experiences.

01 Mar 2025·JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Acquired hemophilia A: a narrative review and management approach in the emicizumab era

Review

Author: Ellsworth, Patrick ; Ma, Alice ; Chen, Sheh-Li ; Ma, Alice D ; Key, Nigel ; Key, Nigel S ; Jones, Lee Ann

Acquired hemophilia A (AHA) is a rare bleeding disorder caused by inhibitory autoantibodies to factor (F)VIII. The goals of treatment are 2-fold, namely immunosuppressive therapy to eradicate the inhibitor and hemostatic management to control bleeding. Emicizumab, a bispecific antibody that acts as a FVIIIa-mimetic, has seen growing use in AHA following its approval for congenital hemophilia A. This review provides an overview of the epidemiology, pathophysiology, diagnosis, and treatment of AHA. Registry, trial, and case series data are assimilated and summarized with an emphasis on a standardized approach that integrates the use of emicizumab. With recent registry data suggesting the need to focus on immunosuppression-related mortality in AHA, we provide treatment recommendations in an algorithmic format that have become the standard of care at our institution. These recommendations are intended to minimize hemostatic product usage and potential toxicity related to immunosuppressive therapy while reducing morbidity and rehospitalization rates for bleeding. The proposed treatment algorithm, which includes key interventions by phase of therapy, can be readily implemented at centers that have rapid access to plasma FVIII activity using a one-stage assay. A case is presented to illustrate the proposed diagnostic and management considerations.

166

News (Medical) associated with Emicizumab

07 Feb 2025

·www.novonordisk.com

Novo Nordisk A/S: Once-weekly Mim8 is well-tolerated and efficacious in children living with haemophilia A with and without inhibitors

Phase 3 FRONTIER3 interim analysis data presented at EAHAD 2025 showed that 74.3% of children on once-weekly Mim8 prophylaxis experienced zero treated bleeds Bagsværd, Denmark, 7 February 2025 – Novo Nordisk today announced interim results from the phase 3 FRONTIER3 trial of 70 children (aged 1-11 years old) with haemophilia A with and without inhibitors. The trial initially assessed once-weekly prophylaxis treatment (regular treatment to prevent prolonged and spontaneous bleeding) with investigational Mim8 before giving participants the option to change to once-monthly dosing after 26 weeks. The data were presented at the 18 th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD 2025) in Milan, Italy. The data showed that Mim8 was well-tolerated and efficacious in children with haemophilia A with and without inhibitors. In part one of the FRONTIER3 study, participants with haemophilia A received once-weekly doses of Mim8 administered under the skin for 26 weeks. In part two, participants had the option to continue with once-weekly dosing or move to once-monthly dosing for the remaining 26 weeks. The interim analysis reports on results at the completion of part one of the study, with some preliminary data shared from part two, which continued following this analysis. For part one of the study in children on once-weekly prophylaxis, the estimated mean (average) annualised bleeding rate (ABR) for treated bleeds was 0.53. The median (middle or central value in the data set) ABR was zero; 74.3% of participants had zero treated bleeds. All children with haemophilia A with inhibitors (n=14) reported zero treated bleeds. After completing the initial 26 weeks of the study, 45% of participants chose to move to once-monthly Mim8, and the rest (55%) remained on the once-weekly dose. “Managing haemophilia A in young children can be a complex balancing act of delivering ongoing care, minimising time out of school, and ensuring their physical, emotional and social wellbeing is optimised,” said Professor Johnny Mahlangu, lead investigator and Director of the Haemophilia Comprehensive Care Centre at Charlotte Maxeke Johannesburg Hospital at University of the Witwatersrand in Johannesburg, South Africa. “The FRONTIER3 interim analysis data are encouraging for families with young children and indicate that Mim8 could offer an efficacious, convenient, flexible dosing option for children, helping to reduce the treatment burden so families can live more normal lives.” Patient- and caregiver-reported outcomes data from part one of the FRONTIER3 trial with once-weekly Mim8 were also presented, which indicated that Mim8 may reduce treatment burden and improve physical function and quality of life (QoL) in children living with haemophilia A with or without inhibitors. At week 26, 98% of caregivers said they preferred Mim8 to prior treatment, of which 73% said they ‘very strongly’ preferred Mim8 to prior treatment. Additionally, at week 26, positive improvement trends were observed in the mean physical function score (a measure of a child’s ability to perform everyday physical activities such as running) and mean QoL total score, compared to baseline. “We see the daily challenges faced by children living with haemophilia A and their caregivers. The FRONTIER3 data represent another step forward in our ambition to provide treatment options that place equal focus on safety and efficacy without requiring a compromise on treatment administration and quality of life,” said Ludovic Helfgott, executive vice president for Rare Disease at Novo Nordisk. “Mim8 is designed with the aim to offer treatment flexibility based on individual lifestyles, so it is encouraging to see that patients and caregivers in these analyses prefer Mim8 over their previous treatment. At Novo Nordisk, our commitment to the rare bleeding disorders community compels us to reduce limitations in the lives of children with haemophilia and their families.” Novo Nordisk expects Mim8 regulatory submission 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 bleeding. It is estimated to affect approximately 1,125,000 people worldwide 1 . Due to the nature of haemophilia being an x-linked recessive disorder, it often presents differently in males compared to females, with ~88% of people diagnosed with haemophilia worldwide being male 2,3 . There are different types of haemophilia, which are characterised by the type of clotting factor protein that is defective or missing. Haemophilia A is caused by a missing or defective clotting Factor VIII (FVIII). About Mim8 Mim8 is an investigational Factor VIIIa (FVIIIa) mimetic bispecific antibody designed with the aim to deliver sustained haemostasis for once-weekly, once every two weeks or once-monthly prophylaxis for people living with haemophilia A with and without inhibitors. Administered under the skin, Mim8 bridges Factor IXa and Factor X. This action replaces Factor VIII, which restores the body’s thrombin generation capacity, helping blood to clot. 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 FRONTIER3 trial FRONTIER3 is a phase 3 clinical trial that evaluated the efficacy and safety profile of Mim8 as a prophylaxis treatment in 70 children (aged 1-11 years old) with haemophilia A with inhibitors (n=14) and without inhibitors (n=56) 4 . The study consisted of two parts: In the FRONTIER3 trial, no major safety concerns (deaths, thromboembolic events or severe treatment-emergent adverse events) or clinical evidence of neutralising anti-drug antibodies (ADAs) were observed with Mim8, in line with previous trials. Less than 1% of all injections were reported to have injection site reactions (ISRs). The FRONTIER clinical programme investigates Mim8 as a prophylaxis treatment for people with haemophilia A with or without inhibitors. The phase 3 programme includes FRONTIER2 6 , FRONTIER3 4 , FRONTIER4 7 , and FRONTIER5 8 . 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 76,300 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com , Facebook , Instagram , X , LinkedIn and YouTube . Contacts for further information _______________________ References

Phase 3Clinical Result

07 Feb 2025

·www.novonordisk.com

Novo Nordisk A/S: Once-weekly Mim8 is well-tolerated and efficacious in children living with haemophilia A with and without inhibitors

Phase 3 FRONTIER3 interim analysis data presented at EAHAD 2025 showed that 74.3% of children on once-weekly Mim8 prophylaxis experienced zero treated bleeds Bagsværd, Denmark, 7 February 2025 – Novo Nordisk today announced interim results from the phase 3 FRONTIER3 trial of 70 children (aged 1-11 years old) with haemophilia A with and without inhibitors. The trial initially assessed once-weekly prophylaxis treatment (regular treatment to prevent prolonged and spontaneous bleeding) with investigational Mim8 before giving participants the option to change to once-monthly dosing after 26 weeks. The data were presented at the 18 th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD 2025) in Milan, Italy. The data showed that Mim8 was well-tolerated and efficacious in children with haemophilia A with and without inhibitors. In part one of the FRONTIER3 study, participants with haemophilia A received once-weekly doses of Mim8 administered under the skin for 26 weeks. In part two, participants had the option to continue with once-weekly dosing or move to once-monthly dosing for the remaining 26 weeks. The interim analysis reports on results at the completion of part one of the study, with some preliminary data shared from part two, which continued following this analysis. For part one of the study in children on once-weekly prophylaxis, the estimated mean (average) annualised bleeding rate (ABR) for treated bleeds was 0.53. The median (middle or central value in the data set) ABR was zero; 74.3% of participants had zero treated bleeds. All children with haemophilia A with inhibitors (n=14) reported zero treated bleeds. After completing the initial 26 weeks of the study, 45% of participants chose to move to once-monthly Mim8, and the rest (55%) remained on the once-weekly dose. “Managing haemophilia A in young children can be a complex balancing act of delivering ongoing care, minimising time out of school, and ensuring their physical, emotional and social wellbeing is optimised,” said Professor Johnny Mahlangu, lead investigator and Director of the Haemophilia Comprehensive Care Centre at Charlotte Maxeke Johannesburg Hospital at University of the Witwatersrand in Johannesburg, South Africa. “The FRONTIER3 interim analysis data are encouraging for families with young children and indicate that Mim8 could offer an efficacious, convenient, flexible dosing option for children, helping to reduce the treatment burden so families can live more normal lives.” Patient- and caregiver-reported outcomes data from part one of the FRONTIER3 trial with once-weekly Mim8 were also presented, which indicated that Mim8 may reduce treatment burden and improve physical function and quality of life (QoL) in children living with haemophilia A with or without inhibitors. At week 26, 98% of caregivers said they preferred Mim8 to prior treatment, of which 73% said they ‘very strongly’ preferred Mim8 to prior treatment. Additionally, at week 26, positive improvement trends were observed in the mean physical function score (a measure of a child’s ability to perform everyday physical activities such as running) and mean QoL total score, compared to baseline. “We see the daily challenges faced by children living with haemophilia A and their caregivers. The FRONTIER3 data represent another step forward in our ambition to provide treatment options that place equal focus on safety and efficacy without requiring a compromise on treatment administration and quality of life,” said Ludovic Helfgott, executive vice president for Rare Disease at Novo Nordisk. “Mim8 is designed with the aim to offer treatment flexibility based on individual lifestyles, so it is encouraging to see that patients and caregivers in these analyses prefer Mim8 over their previous treatment. At Novo Nordisk, our commitment to the rare bleeding disorders community compels us to reduce limitations in the lives of children with haemophilia and their families.” Novo Nordisk expects Mim8 regulatory submission 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 bleeding. It is estimated to affect approximately 1,125,000 people worldwide 1 . Due to the nature of haemophilia being an x-linked recessive disorder, it often presents differently in males compared to females, with ~88% of people diagnosed with haemophilia worldwide being male 2,3 . There are different types of haemophilia, which are characterised by the type of clotting factor protein that is defective or missing. Haemophilia A is caused by a missing or defective clotting Factor VIII (FVIII). About Mim8 Mim8 is an investigational Factor VIIIa (FVIIIa) mimetic bispecific antibody designed with the aim to deliver sustained haemostasis for once-weekly, once every two weeks or once-monthly prophylaxis for people living with haemophilia A with and without inhibitors. Administered under the skin, Mim8 bridges Factor IXa and Factor X. This action replaces Factor VIII, which restores the body’s thrombin generation capacity, helping blood to clot. 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 FRONTIER3 trial FRONTIER3 is a phase 3 clinical trial that evaluated the efficacy and safety profile of Mim8 as a prophylaxis treatment in 70 children (aged 1-11 years old) with haemophilia A with inhibitors (n=14) and without inhibitors (n=56) 4 . The study consisted of two parts: In the FRONTIER3 trial, no major safety concerns (deaths, thromboembolic events or severe treatment-emergent adverse events) or clinical evidence of neutralising anti-drug antibodies (ADAs) were observed with Mim8, in line with previous trials. Less than 1% of all injections were reported to have injection site reactions (ISRs). The FRONTIER clinical programme investigates Mim8 as a prophylaxis treatment for people with haemophilia A with or without inhibitors. The phase 3 programme includes FRONTIER2 6 , FRONTIER3 4 , FRONTIER4 7 , and FRONTIER5 8 . 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 76,300 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com , Facebook , Instagram , X , LinkedIn and YouTube . Contacts for further information _______________________ References

Phase 3Clinical Result

07 Feb 2025

·www.novonordisk.com

Novo Nordisk A/S: Once-weekly Mim8 is well-tolerated and efficacious in children living with haemophilia A with and without inhibitors

Phase 3 FRONTIER3 interim analysis data presented at EAHAD 2025 showed that 74.3% of children on once-weekly Mim8 prophylaxis experienced zero treated bleeds Bagsværd, Denmark, 7 February 2025 – Novo Nordisk today announced interim results from the phase 3 FRONTIER3 trial of 70 children (aged 1-11 years old) with haemophilia A with and without inhibitors. The trial initially assessed once-weekly prophylaxis treatment (regular treatment to prevent prolonged and spontaneous bleeding) with investigational Mim8 before giving participants the option to change to once-monthly dosing after 26 weeks. The data were presented at the 18 th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD 2025) in Milan, Italy. The data showed that Mim8 was well-tolerated and efficacious in children with haemophilia A with and without inhibitors. In part one of the FRONTIER3 study, participants with haemophilia A received once-weekly doses of Mim8 administered under the skin for 26 weeks. In part two, participants had the option to continue with once-weekly dosing or move to once-monthly dosing for the remaining 26 weeks. The interim analysis reports on results at the completion of part one of the study, with some preliminary data shared from part two, which continued following this analysis. For part one of the study in children on once-weekly prophylaxis, the estimated mean (average) annualised bleeding rate (ABR) for treated bleeds was 0.53. The median (middle or central value in the data set) ABR was zero; 74.3% of participants had zero treated bleeds. All children with haemophilia A with inhibitors (n=14) reported zero treated bleeds. After completing the initial 26 weeks of the study, 45% of participants chose to move to once-monthly Mim8, and the rest (55%) remained on the once-weekly dose. “Managing haemophilia A in young children can be a complex balancing act of delivering ongoing care, minimising time out of school, and ensuring their physical, emotional and social wellbeing is optimised,” said Professor Johnny Mahlangu, lead investigator and Director of the Haemophilia Comprehensive Care Centre at Charlotte Maxeke Johannesburg Hospital at University of the Witwatersrand in Johannesburg, South Africa. “The FRONTIER3 interim analysis data are encouraging for families with young children and indicate that Mim8 could offer an efficacious, convenient, flexible dosing option for children, helping to reduce the treatment burden so families can live more normal lives.” Patient- and caregiver-reported outcomes data from part one of the FRONTIER3 trial with once-weekly Mim8 were also presented, which indicated that Mim8 may reduce treatment burden and improve physical function and quality of life (QoL) in children living with haemophilia A with or without inhibitors. At week 26, 98% of caregivers said they preferred Mim8 to prior treatment, of which 73% said they ‘very strongly’ preferred Mim8 to prior treatment. Additionally, at week 26, positive improvement trends were observed in the mean physical function score (a measure of a child’s ability to perform everyday physical activities such as running) and mean QoL total score, compared to baseline. “We see the daily challenges faced by children living with haemophilia A and their caregivers. The FRONTIER3 data represent another step forward in our ambition to provide treatment options that place equal focus on safety and efficacy without requiring a compromise on treatment administration and quality of life,” said Ludovic Helfgott, executive vice president for Rare Disease at Novo Nordisk. “Mim8 is designed with the aim to offer treatment flexibility based on individual lifestyles, so it is encouraging to see that patients and caregivers in these analyses prefer Mim8 over their previous treatment. At Novo Nordisk, our commitment to the rare bleeding disorders community compels us to reduce limitations in the lives of children with haemophilia and their families.” Novo Nordisk expects Mim8 regulatory submission 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 bleeding. It is estimated to affect approximately 1,125,000 people worldwide 1 . Due to the nature of haemophilia being an x-linked recessive disorder, it often presents differently in males compared to females, with ~88% of people diagnosed with haemophilia worldwide being male 2,3 . There are different types of haemophilia, which are characterised by the type of clotting factor protein that is defective or missing. Haemophilia A is caused by a missing or defective clotting Factor VIII (FVIII). About Mim8 Mim8 is an investigational Factor VIIIa (FVIIIa) mimetic bispecific antibody designed with the aim to deliver sustained haemostasis for once-weekly, once every two weeks or once-monthly prophylaxis for people living with haemophilia A with and without inhibitors. Administered under the skin, Mim8 bridges Factor IXa and Factor X. This action replaces Factor VIII, which restores the body’s thrombin generation capacity, helping blood to clot. 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 FRONTIER3 trial FRONTIER3 is a phase 3 clinical trial that evaluated the efficacy and safety profile of Mim8 as a prophylaxis treatment in 70 children (aged 1-11 years old) with haemophilia A with inhibitors (n=14) and without inhibitors (n=56) 4 . The study consisted of two parts: In the FRONTIER3 trial, no major safety concerns (deaths, thromboembolic events or severe treatment-emergent adverse events) or clinical evidence of neutralising anti-drug antibodies (ADAs) were observed with Mim8, in line with previous trials. Less than 1% of all injections were reported to have injection site reactions (ISRs). The FRONTIER clinical programme investigates Mim8 as a prophylaxis treatment for people with haemophilia A with or without inhibitors. The phase 3 programme includes FRONTIER2 6 , FRONTIER3 4 , FRONTIER4 7 , and FRONTIER5 8 . 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 76,300 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com , Facebook , Instagram , X , LinkedIn and YouTube . Contacts for further information _______________________ References

Phase 3Clinical Result

100 Deals associated with Emicizumab

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External Link

KEGG	Wiki	ATC	Drug Bank
D10821	Emicizumab	B02BX06	DB13923

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Coagulation Protein Disorders	Japan	Chugai Pharmaceutical Co., Ltd.	23 Mar 2018
Hemophilia A	United States	Genentech, Inc.	16 Nov 2017

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Factor 8 Deficiency, Acquired	Phase 2	Austria	Hoffmann-La Roche, Inc.	23 Mar 2021
Factor 8 Deficiency, Acquired	Phase 2	Germany	Hoffmann-La Roche, Inc.	23 Mar 2021
Von Willebrand Disease, Type 3	Phase 1	United States	Genentech, Inc.	01 Nov 2022
Hemophilia	Phase 1	China	Roche China Holding Ltd.	12 Mar 2018
Hemophilia	Phase 1	China	F. Hoffmann-La Roche Ltd.	12 Mar 2018

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Hemophilia A	-	Emicizumab	zqebjopieh(cbypuiswcu) = awrrcnomnp fhvtfmiido (iatgkurjyt, 1.1) View more	-	09 Dec 2024
NCT04431726 (HAVEN 7, ASH2024)	Phase 3	Hemophilia A FVIII inhibitors	55	Emicizumab	hjdrzmyxoi(vhiqlocmzn) = cwivpltdgx phcapozrpp (agfrcidluz )	Positive	08 Dec 2024
				Factor VIII	hjdrzmyxoi(vhiqlocmzn) = ohegyxfpwl phcapozrpp (agfrcidluz )
ASH2024	Not Applicable	-	-	Emicizumab	ynlnsypaht(bmtldqpkea) = kzwroaiorw cltavqfpkx (faujjntlon ) View more	-	08 Dec 2024
ASH2024	Not Applicable	Hemophilia A	-	Emicizumab	jpymrmmhpp(hjyknnucni) = mfembljwda lmwflbzrrg (pvbatztccx )	-	07 Dec 2024
				Comparators	jpymrmmhpp(hjyknnucni) = vczdfernxq lmwflbzrrg (pvbatztccx )
EHA2024	Not Applicable	Hemophilia A	16	Emicizumab	lpykqnlqnn(wtaewtdrhw) = uupvrjxkkl kyltizfdfl (onwjwdwtgq )	Negative	14 May 2024
				FVIII replacement	lpykqnlqnn(wtaewtdrhw) = knfwiafbsf kyltizfdfl (onwjwdwtgq )
EHA2024	Not Applicable	-	25	octocog alfa	nlawuhfcbe(szwjphccge) = nuiqyhovzg olwzfqaask (bvazemswhl, 38)	-	14 May 2024
				emicizumab	nlawuhfcbe(szwjphccge) = lijiatsgvz olwzfqaask (bvazemswhl, 9)
P11-5.023 (AAN2024)	Not Applicable	Factor 8 Deficiency, Acquired Factor VIII level \| Factor VIII inhibitor level	-	Emicizumab	ovwlnfkqii(drvadlctvl) = Apixaban was initiated for secondary prevention with no ischemic or hemorrhagic events at 3 and 6 month follow ups vfonxynjgk (nwlayhkocq ) View more	Negative	09 Apr 2024
NCT03191799 (STASEY, Pubmed \| TH Open) Manual	Phase 3	Hemophilia A	56	Emicizumab prophylaxis with additional hemostatic medication	wzfiozjble(rmghiwqxjz) = mhwsuxagpn zcnepsacbl (bvjkpwnwzx ) View more	Positive	12 Jan 2024
				Emicizumab prophylaxis without additional hemostatic medication	wzfiozjble(rmghiwqxjz) = ogmctmrbfy zcnepsacbl (bvjkpwnwzx )
ASH2023	Not Applicable	Hemophilia A	-	Emicizumab	keibtfcrod(nvgeuhmejg) = dxkmppgmtb dhyehpefih (oxwsbtnzws )	-	11 Dec 2023
				Efanesoctocog alfa	keibtfcrod(nvgeuhmejg) = dcvpjfavrb dhyehpefih (oxwsbtnzws )
ASH2023	Not Applicable	Hemophilia A	127	Emicizumab prophylaxis	zpbmwlrkxe(glvlpzaxbq) = pbzoramgwf kzrabykojy (pkqwqlybhc ) View more	-	10 Dec 2023

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