Delving into the Latest Updates on Moroctocog alfa with Synapse

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.

01 Oct 2023·Indian Journal of Hematology and Blood Transfusion

Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Patients with Hemophilia A in India

Article

Author: Rangarajan, Savita ; Gupta, Pritam ; Ramakanth, G S H ; Rupon, Jeremy ; Chand, Rohit ; Muley, Hitesh Bhaskarrao ; Pai, Seema ; Choraria, Nirmalkumar ; John, M Joseph ; Apte, Shashikant ; Parvatini, Shyam ; Simoneau, Damien ; Chhabra, Amit

AbstractPurposeHemophilia A is an X-linked congenital disorder, characterized by factor VIII (FVIII) deficiency. Globally, India has the highest population of patients with hemophilia, and there is a clear unmet need for appropriate and effective treatment for this patient population. This multicenter, open-label, post-approval study evaluated the safety and efficacy of moroctocog alfa in patients with moderate or severe congenital hemophilia A in India.MethodsIntravenous moroctocog alfa was administered 30 ± 5 IU/kg 3 times weekly for bleeding prophylaxis, according to the local product document. Participants were treated for up to 8 weeks, with an up to 4-week screening period and a subsequent post-treatment, 28-day safety observation period. Patients continued in the study until at least 24 exposure days or a period of up to 8 weeks on moroctocog alfa.ResultsA total of 50 participants were enrolled, and 48 (85.7%) completed the study. No participants developed FVIII inhibitors during the study. The mean (SD) annualized bleeding rate during moroctocog alfa prophylaxis was 0.79 (2.0) with a median (range) of 0.00 (0.0, 6.8). The mean (SD) annualized total factor consumption (TFC) per participant was 287,432 (93,866) IU; the mean (SD) annualized TFC by weight per participant was 4176 (858) IU/kg. Moroctocog alfa was well tolerated with no reported treatment-emergent adverse event-related dose reductions, discontinuations, or serious adverse events.ConclusionMoroctocog alfa was safe, effective, and well tolerated in Indian participants with congenital moderate to severe hemophilia A. No participant developed FVIII inhibitors during the study.

100 Deals associated with Moroctocog alfa

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KEGG	Wiki	ATC	Drug Bank
D08232	Moroctocog alfa	B02BD02	DB13999

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Von Willebrand Diseases	Australia	Pfizer Australia Pty Ltd.	26 Jun 2009
Hemophilia A	Liechtenstein	Pfizer Europe MA EEIG	13 Apr 1999
Hemophilia A	European Union	Pfizer Europe MA EEIG	13 Apr 1999
Hemophilia A	Iceland	Pfizer Europe MA EEIG	13 Apr 1999
Hemophilia A	Norway	Pfizer Europe MA EEIG	13 Apr 1999

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Atrial Fibrillation	NDA/BLA	-	Wyeth AB	20 May 2002
Hemophilia A	NDA/BLA	United States	Genetics Institute LLC	27 Jun 2001
Hemophilia A	Phase 1	United States	Genetics Institute LLC	27 Jun 2001

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Clinical Result

Indication

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EHA2021	Not Applicable	Hemophilia A	-	Moroctocog alpha	rohnfbbkma(xvchzuflas) = idgnkbufvp gchbxiwfnk (wurpgxyysd )	-	09 Jun 2021
EHA2021	Not Applicable	Hemophilia A	-	Plasma FVIII and Octocog alpha	rohnfbbkma(xvchzuflas) = tuxrkeoikt gchbxiwfnk (wurpgxyysd )	-	09 Jun 2021
REF.IT Registry (ISTH2019)	Not Applicable	Hemophilia A	27	Moroctocog-alpha	rjyayeuadq(ymxikmnndj) = suzjzkjuox feijqbixto (rexbcxbkdk ) 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)	waulomjmaz(awozjjsejy) = bayfjvirda catekwpjaw (guklqjdccn, rozfegmtke - zcrbpheunj)	-	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)	waulomjmaz(awozjjsejy) = pmxrdxmuhi catekwpjaw (guklqjdccn, eglnfjzgin - xrmohwhosf) View more	-	11 Jan 2019
NCT02461992 (Pubmed \| Med Lett Drugs Ther) Manual	Phase 1	Hemophilia A	13	Moroctocog Alfa	jwmjdmocod(cqjtokpgtm) = enrbmexsnq vmgcsiwwjg (jcbaamtsys ) View more	Positive	01 Jul 2017
NCT02461992 (CTgov)	Phase 1	Hemophilia A	13	Xyntha	ewpfsvyouq(vwfgwjzkol) = oktgzoyvxz ttrmmjnxne (kgyrbuxqld, bvyevgupbs - dxwiesnxhs) View more	-	15 Jun 2016
NCT00868530 (CTgov)	Phase 3	Hemophilia A	53	Xyntha	fleuohltpj(ygywddncgv) = amwpgvhyti agonfyhbtx (flokxuugsd, ehqhcsbfmg - brmsvfjhey) View more	-	08 Feb 2011
NCT00759655 (CTgov)	Phase 3	Hemophilia A	1	Xyntha	vyxiuzdyul(ngsbzdknhq) = qxqjofkjbv cidfgbaoeh (aupkwgjrvn, ijyhqqdukg - zjzxkntaal) View more	-	04 Jan 2011
NCT00243659 (CTgov)	Phase 3	Hemophilia A	30	Refacto AF (Bolus Injection)	pxamptcmdm(mjnusxoueh) = uzazlasrns hnexsomgxr (wfuftweipc, zfuiaknnxh - azeazgejnt) View more	-	20 Jul 2009
NCT00243659 (CTgov)	Phase 3	Hemophilia A	30	Refacto AF (Continuous Infusion)	pxamptcmdm(mjnusxoueh) = ogzhmxzsde hnexsomgxr (wfuftweipc, otieddmbgb - kwdresdphm) View more	-	20 Jul 2009

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