Delving into the Latest Updates on Nonacog alfa with Synapse

Overview

Basic Info

Drug Type

Recombinant coagulation factor

Synonyms

Benefix, Benefix Rts, Blood-coagulation-factor-IX-synthetic-human, Factor-IX-Genetics-Institute

+ [9]

Target

F8

Action

stimulants

Mechanism

F8 stimulants(Coagulation factor VIII stimulants), Blood coagulation pathway activation

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication

Hemophilia B

Inactive Indication-

Originator Organization

Pfizer Inc.

Active Organization

Pfizer Europe MA EEIG

Haleon (Suzhou) Pharmaceutical Co. Ltd.

Pfizer Japan, Inc.

+ [2]

Inactive Organization

Pfizer Inc.Wyeth AB

Drug Highest PhaseApproved

First Approval Date

United States (11 Feb 1997),

Hemophilia B

RegulationSpecial Review Project (China), Orphan Drug (Japan)

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Structure/Sequence

Sequence Code 23878

Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B. The current single country, multi-centric, open label, non-randomized clinical trial is a post-approval study to fulfill the Central Drugs Standard Control Organization (CDSCO) request for supplementary information relating to the use of nonacog alfa in Indian subjects with hemophilia B.

Start Date10 Feb 2020

Sponsor / Collaborator

Pfizer Inc.

NCT03186677

/ CompletedPhase 1

A Phase 1, Open-label, Multi-center, Dose-escalation Study to Investigate the Safety, Pharmacokinetics and Pharmacodynamics of ISU304 in Previously Treated Hemophilia B Patients

This study is a phase 1, open-label, multi-center, dose-escalation study to investigate the safety, pharmacokinetics and pharmacodynamics of ISU304/CB2679d in previously treated hemophilia B patients.

Start Date03 Jun 2017

Sponsor / Collaborator

ISU Abxis Co., Ltd.

[+1]

100 Clinical Results associated with Nonacog alfa

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100 Translational Medicine associated with Nonacog alfa

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100 Patents (Medical) associated with Nonacog alfa

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80

Literatures (Medical) associated with Nonacog alfa

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

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 & blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion

Nonacog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Patients with Moderately Severe or Severe Hemophilia B in India

Article

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

Abstract:

Purpose:

Hemophilia B is an X-linked congenital bleeding disorder caused by a deficiency of coagulation factor IX (FIX) clotting activity. This study evaluated safety and efficacy of nonacog alfa, a recombinant human blood coagulation FIX replacement product, in males aged 12–65 years with hemophilia B (FIX activity ≤ 2%) with or without inhibitors in India.

Methods:

In this multicenter, open-label, post-approval phase 4 study, participants were treated for up to 8 weeks, with up to a 4-week screening period and a subsequent post-treatment 28-day safety observation period. Intravenous nonacog alfa 40 IU/kg (range 13–78 IU/kg) was administered at intervals of 3–4 days, in accordance with the approved local product document.

Results:

A total of 25 participants were enrolled and completed the study. No participants developed FIX inhibitors during the study, experienced treatment-related adverse events (AEs) or serious AEs, or developed a thrombotic event and/or hypersensitivity reaction. No participants experienced bleeding events requiring on-demand treatment with nonacog alfa. Seventeen bleeding episodes (16 spontaneous and 1 traumatic) were reported in 10 participants; all occurred post treatment, with the exception of a minor gum-bleeding event, and were managed without treatment. The mean (SD) annualized total factor consumption (TFC) per patient was 224,582 (75,527) IU; the mean (SD) annualized TFC by weight per patient was 3639 (573) IU/kg.

Conclusion:

Nonacog alfa was safe and effective for the prevention of hemorrhagic episodes in Indian males with congenital, severe hemophilia B. No participants developed FIX inhibitors, and no new safety signals were reported.

01 Mar 2023·Research and practice in thrombosis and haemostasis

Treatment switch to nonacog beta pegol factor IX in hemophilia B: A Canadian cost-consequence analysis based on real-world factor IX consumption and clinical outcomes.

Article

Author: Bentley, Anthony ; Matino, Davide ; Christoffersen, Pia ; Germini, Federico ; Iorio, Alfonso ; Poon, Man-Chiu ; Luckevich, Maria D ; Caillaud, Alexandre ; MacDonald, Vance ; Iserman, Emma ; Keepanasseril, Arun

Background:

The Canadian Bleeding Disorders Registry (CBDR) is a source of real-world data for Canadian patients with hemophilia B. Nonacog beta pegol (N9-GP), an extended half-life (EHL) recombinant factor IX (FIX) concentrate, was awarded a Canadian Blood Services contract in 2018 and subsequently made available across Canada (except Québec) to adult patients. For most patients already on another EHL FIX treatment, a switch to N9-GP occurred.

Objectives:

This study estimates the impact on treatment costs of a switch from a prior FIX to N9-GP based on annualized bleed rates and FIX consumption volumes before and after N9-GP switch from the CBDR.

Methods:

Real-world data from the CBDR for total FIX consumption and annualized bleed rates were used to inform a deterministic 1-year cost-consequence model. The model considered that the EHL to N9-GP switches were from eftrenonacog alfa and the standard half-life switches were from nonacog alfa. Because FIX prices are confidential in Canada, the model assumed cost parity for annual prophylaxis with each FIX based on the product monograph recommended dosing regimen to calculate an estimated price per international unit for each product.

Results:

The switch to N9-GP resulted in improvements in real-world annualized bleed rates and therefore reductions in annual breakthrough bleed treatment costs. Switching to N9-GP also resulted in reduced real-world annual FIX consumption for prophylaxis. Overall, annual treatment costs were 9.4% and 10.5% lower after the switch to N9-GP from nonacog alfa and eftrenonacog alfa, respectively.

Conclusion:

N9-GP improves clinical outcomes and may be cost-saving vs nonacog alfa and eftrenonacog alfa.

100 Deals associated with Nonacog alfa

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External Link

KEGG	Wiki	ATC	Drug Bank
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R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemophilia B	United States	Wyeth Pharmaceuticals LLC	11 Feb 1997

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04286412 (CTgov)	Phase 4	Hemorrhage \| Hemophilia B	25	Nonacog Alfa	qaqmhkbxeu = lcgzajfzro poejsalhhn (kqzrebscyt, equkyhacev - vqqumvafiw) View more	-	30 Jul 2021
NCT02336178 (Pubmed \| Medicine (Baltimore)) Manual	Phase 4	Hemophilia B	70	Nonacog Alfa	qdmgkxxnox(tchwjcupxh) = zdlsxyvkkf pwcasczvwx (scayopmewd, 0 - 7.7) View more	Positive	28 May 2021
NCT03091751 (CTgov)	Phase 2	Hemophilia B	13	BeneFIX	pjmzmftfiz(qqfximktjb) = ixqrjydikz bddjckiqsf (ohghcfhjhp, 563.081) View more	-	25 Jul 2017
NCT02336178 (CTgov)	Phase 4	Hemophilia B	70	Benefix	kkbwhcgofy(zreusbihqe) = abzjmjsrua kgydmhphuh (cujgedoyyk, fkgbfquhzm - knwxecsltk) View more	-	04 May 2017
NCT02213250 (CTgov)	Phase 1	Hemophilia B	12	BeneFIX (BeneFIX 50 IU/kg; >=6 and <12 Years)	tmzblzvjiz(bfmogfyity) = xsuekucoii uajtbwnorj (kvlfolmwto, 26) View more	-	25 Jul 2016
NCT02213250 (CTgov)	Phase 1	Hemophilia B	12	BeneFIX (BeneFIX 50 IU/kg; Age Group:>=12 Years)	tmzblzvjiz(bfmogfyity) = cmuwteskrw uajtbwnorj (kvlfolmwto, 16) View more	-	25 Jul 2016
NCT00364182 (CTgov)	Phase 3	Hemophilia B	50	BeneFIX (BeneFIX OD1)	nxjwarwevc(qnvszzuiwg) = xytuhfrmol opxrmkmcdi (ijpyarrtsm, kzxqipzdzt - rkzfzkapwz) View more	-	23 Aug 2011
NCT00364182 (CTgov)	Phase 3	Hemophilia B	50	BeneFIX (BeneFIX 100 IU/kg)	nxjwarwevc(qnvszzuiwg) = qjmifbtini opxrmkmcdi (ijpyarrtsm, yaszxbhmsu - wsjqmzougy) View more	-	23 Aug 2011
NCT00866606 (CTgov)	Phase 3	Hemophilia B	35	BeneFIX	zmsxvsvexv(pigikighbs) = ilhrgeecgz isnkqiovqs (qtvnaxovxd, 0.72) View more	-	04 Jan 2011
NCT00749476 (CTgov)	Phase 4	Hemophilia B	1	BeneFIX	mxzkeqkdbb(fmzqgsobjb) = ujiybqoesa znijrjqpms (gngcuggkwq, tzpompcbax - qnsydmuies) View more	-	29 Jun 2010

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