Delving into the Latest Updates on Moroctocog alfa with Synapse

Overview

Basic Info

Drug Type

Recombinant coagulation factor

Synonyms

Antihemophilic Factor (Recombinant)(Genetics Institute), BDDrFVIII, FuseNGO

+ [14]

Target

F10

Mechanism

F10 stimulants(Coagulation factor X stimulants), Blood coagulation pathway activation, Coagulation factor VIII replacements

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication

Von Willebrand DiseasesHemophilia A

Inactive Indication

Atrial Fibrillation

Originator Organization

Pfizer Inc.

Active Organization

Pfizer Australia Pty Ltd.Pfizer Europe MA EEIGWyeth Pharmaceuticals LLC

Inactive Organization

Wyeth AB

Genetics Institute LLC

Drug Highest PhaseApproved

First Approval Date

EU (13 Apr 1999),

Hemophilia A

RegulationPriority Review (CN)

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Structure/Sequence

Sequence Code 9955158

Source: *****

Sequence Code 139887

Source: *****

Hemophilia is caused by the deficiency of clotting factors due to a single genetic abnormality. Replacement therapies have evolved from plasma-derived to recombinant coagulation factor concentrates but continue to have certain limitations. Monoclonal antibodies are clinical prophylactic treatment options unaffected by inhibitors and have better compliance than coagulation factor concentrates for patients with hemophilia. Gene therapy is a breakthrough in hemophilia treatment, as it drives the hepatic expression of factor VIII or factor IX and requires only a single administration to enable long-term replacement treatment in adult patients. Furthermore, biopharmaceutical products that target new pathways unaffected by inhibitors, including tissue factor pathway inhibitors, activated protein C, and antithrombin, as well as pharmaceutical technology advances to reduce dosing frequency, have demonstrated promising clinical results. This review provides a comprehensive overview of these biopharmaceutical products and explores the future of hemophilia treatment.

01 Nov 2024·Gene Therapy

Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice

Article

Author: Zhang, Feixu ; Wu, Xia ; Hua, Baolai ; Xiao, Xiao ; Zhou, Xinyue ; He, Xinyi ; Li, Zhanao

In hemophilia, deficiency of factor VIII or IX prevents the activation of the common coagulation pathway, and inhibits the conversion of FX to activated FXa, which is required for thrombin generation. We hypothesized that the direct expressed FXa has the potential to activate the common pathway and restore coagulation in hemophilia patients. In this study, the cassettes that expressed FXa, FXaop and FXa-FVII were packaged into an engineered AAV capsid, AAV843, and were delivered into hemophilia A and B mice by intravenous injection. AAV-FXaop could be stably expressed in vivo and showed the best immediate and prolonged hemostatic effects, similar to those of commercial drugs (Xyntha and Benefix). AAV-FXaop also significantly inhibited bleeding in hemophilia A mice with inhibitors. In addition, FXa expression in joints significantly alleviated the occurrence of hemophilic synovitis. AAV-delivered FXa may be a novel target for treating hemophilic and hemophilic synovitis.

30 Jul 2024·Zhonghua yi xue za zhi

[Long-term health-related quality of life and related factors in children with severe hemophilia A who received regular low-dose prophylaxiss].

Article

Author: Zhao, Y Q ; Zhou, Y ; Li, K X ; Zhang, S N ; Wang, S J ; Li, Z ; Xiao, J

Objective: To investigate long-term health-related quality of life (HRQoL) and related factors in children with severe hemophilia A (HA) who received regular low-dose prophylaxis. Methods: Clinical data of severe HA children who began to receive regular low-dose coagulation factor Ⅷ (FⅧ) prophylaxis in Peking Union Medical College Hospital from January 1, 2008 to December 31, 2011 were retrospectively enrolled. The longest last follow-up period was May 31, 2023. The attendance of school or work and daily physical activity during the last follow-up were investigated. The patients were divided into full attendance group and incomplete attendence group according to attendance. The patients were divided into into exercise attainment group (reached Chinese sports recommendation) and exercise nonattainment group according to the exercise status. Barthel score was used to assess activities of daily living and Haemo-QoL was used to assess quality of life. Long-term HRQoL for children aged 8-16 years and patients aged 17 years and above were assessed using Haemo-QoL SF and Haem-A-QoL versions, respectively. Spearman correlation analysis was used to examine the correlation between treatment conditions and Haemo-QoL scores. Results: A total of 22 cases were enrolled, the prophylaxis initiation age ranged from 1.8-17.9 (10.4±3.8) years old. The average prophylactic FⅧ dose during low-dose prophylaxis was 24.2 U/kg per week and the follow-up time was 6.3-15.1 (9.6±2.8) years. At the last follow-up, the age of the patients was (20.2±5.4) years, of which 14 (63.6%) were adults over 18 years old. There were 15 patients in the full attendance group and 7 patients in the incomplete attendence group. Compared with the full attendance group, the incomplete attendence group had a smaller preventive treatment dose [M(Q_1, Q₃), (28.4±11.1) vs (15.3±3.7) U/kg, P=0.012], shorter preventive treatment time [148. 1 (18.6, 346.5) vs 48.0 (32.0, 156.9) weeks, P=0.017], and higher annual joint bleeding rate (AJBR) [12.5 (6.0, 22.3) vs 14.2 (13.2, 17.8) times, P=0.017]. There were 7 cases in the exercise attainment group and 15 cases in the exercise nonattainment group. Compared to the exercise attainment group, the exercise nonattainment group had shorter preventive treatment time[313. 7 (156.9, 366.0) vs 48.0 (16.5, 108.9) weeks, P=0.006], a higher AJBR [7.0 (5.1, 10.0) vs 23.3 (12.5, 29.8), P=0.003] and a higher hemophilia joint health score (HJHS) [9.0 (2.0, 15.5) vs 23.0 (12.0, 27.8), P=0.014]. Barthel score showed 81.8% (18 cases) of the patients' living ability was not influenced by the illness. In Haemo-QoL score, the total score of Haemo-QoL SF in 7 cases was (47.6±17.0) scores, the total score of Haem-A-QoL in 15 cases was (45.2±22.6) scores. The daily activity dimension of the Haem-A-QoL score was the lowest [38.2 (10.9, 45.5) scores], which was positively correlated with the starting age of prophylactic initiation (r=0.501, P=0.057), and negatively correlated with the duration of prophylaxis (r=-0.545, P=0.036). Conclusions: Regular low-dose prophylaxis could improve the long-term HRQoL of some children with severe HA, and children with higher prophylactic doses and longer prophylactic treatment time have higher quality of life.

100 Deals associated with Moroctocog alfa

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External Link

KEGG	Wiki	ATC	Drug Bank
D08232	Moroctocog alfa	B02BD02	DB13999

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Von Willebrand Diseases	AU	Pfizer Australia Pty Ltd.	26 Jun 2009
Hemophilia A	IS	Pfizer Europe MA EEIG	13 Apr 1999
Hemophilia A	EU	Pfizer Europe MA EEIG	13 Apr 1999
Hemophilia A	NO	Pfizer Europe MA EEIG	13 Apr 1999
Hemophilia A	LI	Pfizer Europe MA EEIG	13 Apr 1999

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hemophilia A	Phase 1	US	Genetics Institute LLC	27 Jun 2001
Hemophilia A	Preclinical	US	Genetics Institute LLC	27 Jun 2001
Atrial Fibrillation	Discovery	-	Wyeth AB	20 May 2002

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EHA2021	Not Applicable	Hemophilia A	-	Moroctocog alpha	fsctsyutuk(vhmssolkqy) = omocteqgmp lusjgvotsk (hhncveghvs )	-	09 Jun 2021
EHA2021	Not Applicable	Hemophilia A	-	Plasma FVIII and Octocog alpha	fsctsyutuk(vhmssolkqy) = sqilernmvl lusjgvotsk (hhncveghvs )	-	09 Jun 2021
REF.IT Registry (ISTH2019)	Not Applicable	Hemophilia A	27	Moroctocog-alpha	dczjfxxujx(mvrhzxkgtv) = djnoorwbhm itkmrubsea (fohxbdhrki ) View more	Positive	10 Jun 2019
NCT00543439 (CTgov)	Phase 3	Hemophilia A	66	Moroctocog Alfa (AF-CC) (Moroctocog Alfa (AF-CC), On Demand Cohort: On Demand Therapy)	dccmhhlzxa(ahkwwvqbzv) = drznemjsdx crsdzjbmyt (dpamnrtikc, hyahtvwzsq - rteisbbroo)	-	11 Jan 2019
NCT00543439 (CTgov)	Phase 3	Hemophilia A	66	RP+Moroctocog Alfa (AF-CC) (Moroctocog Alfa (AF-CC), On Demand Cohort: RP Therapy 25 IU/kg)	dccmhhlzxa(ahkwwvqbzv) = vjjxjwngdq crsdzjbmyt (dpamnrtikc, awuokcmyac - qopczvubyl) View more	-	11 Jan 2019
NCT02461992 (Pubmed \| Med Lett Drugs Ther) Manual	Phase 1	Hemophilia A	13	Moroctocog Alfa	ssmuvwtuaa(aahpkkpkaf) = dfvnrfhsvs ebfsxyvoxd (gizxhmkvmz ) View more	Positive	01 Jul 2017
NCT02461992 (CTgov)	Phase 1	Hemophilia A	13	Xyntha	yrqvchuhip(dzpoumkrif) = dtwffiufym cmqkokggpp (zkhtnwpvnc, xtwrqfptap - earspflpst) View more	-	15 Jun 2016
NCT00868530 (CTgov)	Phase 3	Hemophilia A	53	Xyntha	rstpgwiiuo(llehdyxjes) = kzlbjwaehm zrvgpjxvig (rktquamxxw, irijjjptso - gbnmvyjfwm) View more	-	08 Feb 2011
NCT00759655 (CTgov)	Phase 3	Hemophilia A	1	Xyntha	vosuqzffsi(cwnvstrfrs) = hjzoxunuce ubudbxzthf (xnrkvqxnal, sqyqiuwshc - kwccywomty) View more	-	04 Jan 2011
NCT00243659 (CTgov)	Phase 3	Hemophilia A	30	Refacto AF (Bolus Injection)	kvzrfyrtro(xdpcjolbvs) = okqhfiwndx pgmogujzss (eqdqmbcdcv, ksrkmarguf - oqhfedxyxg) View more	-	20 Jul 2009
NCT00243659 (CTgov)	Phase 3	Hemophilia A	30	Refacto AF (Continuous Infusion)	kvzrfyrtro(xdpcjolbvs) = adjqqrazzq pgmogujzss (eqdqmbcdcv, fhocxtngjq - vsftspslzk) View more	-	20 Jul 2009