Delving into the Latest Updates on Nonacog beta pegol with Synapse

Overview

Basic Info

Drug Type

Recombinant coagulation factor

Synonyms

Coagulation Factor IX (Recombinant), GlycoPEGylated, Glycopegylated rFIX, La-rFIX derivative

+ [14]

Target

factor IX

Mechanism

factor IX stimulants(Coagulation factor IX stimulants)

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication

Hemophilia BHemorrhage

Inactive Indication-

Originator Organization

Novo Nordisk A/S

Active Organization

Novo Nordisk A/SNovo Nordisk Pharmaceuticals Pty Ltd.Novo Nordisk, Inc.

+ [1]

Inactive Organization-

Drug Highest PhaseApproved

First Approval Date

US (31 May 2017),

Hemophilia B

RegulationOrphan Drug (US)

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Gene Sequence

Sequence Code 207242

Source: *****

The study investigates how well the medicine called nonacog beta pegol (N9-GP) works in Chinese people with haemophilia B. Participants will be treated with N9-GP. This is a medicine that doctors can already prescribe in other countries. The medicine will be injected into a vein (intravenous injection). At the visits to the clinic, the medicine will be injected by the study doctor. When treating themselves at home, participants inject the medicine using a needle and vial set. The study will last for about 12-16 months. The participants will have between 9 and 19 visits to the clinic and possibly also some phone calls with the study doctor. At all visits to the clinic, the participants will have blood samples taken.

Start Date18 May 2022

Sponsor / Collaborator

Novo Nordisk A/S

NCT03875547 / Enrolling by invitationNot Applicable

Post-marketing Surveillance (Use Result Surveillance) With Refixia®. A Multicentre, Non-interventional Post Marketing Surveillance of Safety and Effectiveness of Refixia® in Routine Clinical Care With Haemophilia B Patients in Japan.

The participants are invited to take part in this study because they have Haemophilia B. The purpose of this study is to assess the safety and effectiveness of Refixia® about long-term routine use in patients with Haemophilia B. The participants will get Refixia® as prescribed to them by their study doctor. The study will last up to Sep 2025 for the participant. The participants may be asked to fill in the quality of life questionnaires (if they are above age of 15). The blood samples taken from the participants as part of routine clinical practice will also be used to investigate the safety for the long-term use of Refixia®.

Start Date23 Aug 2019

Sponsor / Collaborator

Novo Nordisk A/S

NCT03745924 / Enrolling by invitationNot Applicable

A Non-Interventional Post-Authorisation Safety Study (PASS) in Male Haemophilia B Patients Receiving Nonacog Beta Pegol (N9-GP) Prophylaxis Treatment

This study will collect information on side effects and how well Refixia/REBINYN works during long-term treatment (prophylaxis) in males with haemophilia B. While taking part in this study, participants will receive the same treatment as given to them by their study doctor. All visits at the clinic are done in the same way as the participants are used to. During visits at the clinic, participants might be asked for some relevant tests if considered useful by their study doctor. During the visits, the participants study doctor might ask if the participants had any side effects since their last study visit. The participants will be asked to note down the number of bleeds and the treatment of their bleeds as well as their regular prophylaxis. During the visits to the clinic, the participants will be asked to answer some questionnaires about their quality of life and their ability to be physically active. The participant's participation in the study will last for 4-9 years, depending on when they join the study. Participants are free to leave the study at any time and for any reason. This will not affect their current and future medical care.

Start Date01 Apr 2019

Sponsor / Collaborator

Novo Nordisk A/S

100 Clinical Results associated with Nonacog beta pegol

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100 Translational Medicine associated with Nonacog beta pegol

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100 Patents (Medical) associated with Nonacog beta pegol

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41

Literatures (Medical) associated with Nonacog beta pegol

01 Feb 2024·Research and practice in thrombosis and haemostasis

Nonacog beta pegol prophylaxis in children with hemophilia B: safety, efficacy, and neurodevelopmental outcomes for up to 8 years

Article

Author: Chan, Anthony K C ; Walsh, Karin S ; Carcao, Manuel ; Mrakotsky, Christine ; Shapiro, Kevin ; Nielsen, Pernille Højlund ; Holst, Helle

Background:

Nonacog beta pegol (N9-GP) is an extended half-life PEGylated factor (F)IX product with established efficacy and short-term safety in persons with hemophilia B (HB). Long-term safety has been evaluated for polyethylene glycol exposure but not N9-GP.

Objectives:

To assess safety, neurodevelopmental, and efficacy outcomes of children with HB receiving N9-GP prophylaxis across 2 open-label, single-arm, phase 3 studies: paradigm5 (previously treated patients [PTPs]) and paradigm6 (previously untreated patients [PUPs]) in this interim analysis.

Methods:

PTPs (aged ≤12 years) and PUPs (aged <6 years) with severe/moderate (≤2% FIX level) HB were recruited to N9-GP prophylaxis (40 IU/kg once weekly) in paradigm5 and paradigm6, respectively. Safety assessments included FIX inhibitor incidence, adverse events, neurocognitive and neurologic outcomes, polyethylene glycol concentration in plasma, and medical events of special interest. Efficacy endpoints included bleeds, N9-GP hemostatic effect, and FIX consumption.

Results:

Overall, 25 patients in paradigm5 and 50 patients in paradigm6 received N9-GP and were followed for up to 8 and 6 years, respectively. No inhibitory antibodies were reported in PTPs; 4 of the 50 PUPs developed inhibitors. Extensive evaluation revealed no neurocognitive or neurologic concerns with N9-GP use in children during the study period. Across both studies, few adverse events were reported as possibly related to N9-GP. High hemostatic response rate, high treatment adherence, low annualized bleeding rates, and no new target joints were reported.

Conclusion:

These data provide the longest follow-up for an extended half-life FIX and confirm the long-term efficacy of N9-GP prophylaxis in children with HB with no observed neurocognitive or neurologic safety concerns.

01 Feb 2024·International journal of laboratory hematology

High variability in Factor IX one‐stage assay in samples spiked with nonacog beta pegol among different pairs of reagent/detection system

Article

Author: Duboscq, C. ; Baques, A. ; Zirpoli, M. ; Ceresetto, J. ; Rosa, C. ; Arias, M. ; Sueldo, E.

Abstract:

Introduction:

Haemophilia B (HB) is an X‐linked hereditary bleeding disorder characterized by coagulation factor IX (FIX) deficiency. To improve the quality of life of patients and adherence to treatment, recombinant factor concentrates modified to extend their half‐life have been developed, called extended half‐life factors (EHL: extended half‐life). Nonacog beta pegol (N9‐GP) is a glycopegylated recombinant human FIX molecule that has a half‐life of 93 h with a single dose and has shown a higher recovery percentage than other molecules. To diagnose and monitor the treatment of haemophiliac patients, FIX activity is determined with the one‐stage clotting assay (OSA) and/or the chromogenic assay. The objective of this work, carried out in three centres, was to measure the recovery of N9‐PG with 10 different activated partial thromboplastin time (APTT) reagents on three platforms, in samples spiked in vitro with N9‐GP, at four different concentration levels.

Methods:

It was measured the recovery of N9‐GP with 10 different APTT reagents (polyphenol, ellagic acid, silice dioxide, colloidal silica as APTT activator on three platforms, in sample spiked in vitro with N9‐GP.

Results:

The results show heterogeneity in the activity of N9‐GP measured by OSA with the different APTT reagents when the calibrations were performed with the specific calibrator of each coagulometer. A recovery percentage between 87% and 108% was obtained only with polyphenol and ellagic acid as activator in the three platforms evaluated. The other reagents studied overestimate or underestimate, with no clear profile. When a calibration curve was performed with a calibrator prepared from the N9‐GP vial, all APTT reagents met the established recovery requirement.

Conclusion:

APTT reagents with polyphenol or ellagic acid as activator would be the only ones appropriate when using the commercially available OSA with specific calibrator to monitor patients treated with N9‐GP.

01 Oct 2023·Research and practice in thrombosis and haemostasis

Use of nonacog beta pegol during surgery in persons with hemophilia B: a case series

Author: Kühnöl, Caspar D ; Matsushita, Tadashi ; Matino, Davide ; Hegemann, Inga ; Phua, Chai W

Background:

Hemophilia B is a coagulation disorder that puts patients at an increased risk of bleeding. Factor (F) IX replacement therapy is traditionally used in such cases to maintain hemostasis. Nonacog beta pegol (N9-GP; Refixia) is a glycoPEGylated, extended half-life, recombinant FIX product that has demonstrated safety and efficacy when used to manage persons with hemophilia B.

Key clinical question:

Given the limited real-world evidence, we aimed to explore the role of N9-GP in maintaining hemostasis in persons with hemophilia B undergoing surgery.

Clinical approach:

In this case series, we report real-world clinical experience with N9-GP to maintain hemostasis in persons with hemophilia B undergoing major and minor surgeries.

Conclusion:

The majority of cases presented in this case series had an excellent or very good hemostatic response, with no reported surgical complications related to the use of N9-GP.

100 Deals associated with Nonacog beta pegol

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External Link

KEGG	Wiki	ATC	Drug Bank
D10757	-	B02BD04	DB13933

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemophilia B	US	Novo Nordisk, Inc.	31 May 2017

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hemorrhage	Phase 3	US	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	JP	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	CA	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	FR	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	DE	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	HU	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	IT	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	MY	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	NL	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	MK	Novo Nordisk A/S	27 Apr 2011

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02141074 (paradigm™6, CTgov)	Phase 3	Hemorrhage \| Hemophilia B	54	nonacog beta pegol (Pre-prophylaxis)	brzuqhpsqj(zdvagfaaow) = yhlqwbeete hgudaslwhr (ytgbirtuie, yeekzmfyxb - ofcsimvluf) View more	-	29 Dec 2023
NCT02141074 (paradigm™6, CTgov)	Phase 3	Hemorrhage \| Hemophilia B	54	nonacog beta pegol (Prophylaxis)	brzuqhpsqj(zdvagfaaow) = wcabcrlgkj hgudaslwhr (ytgbirtuie, xnrihqakkk - uiudjymeai) View more	-	29 Dec 2023
ISTH2022	Not Applicable	Hemophilia B	-	N9-GP 40 IU/kg once weekly	wtgzzvtclf(wqquhdlkgr) = ijcawwxarb lpjnhwdxee (kqczohprqs ) View more	-	09 Jul 2022
NCT02141074 (Pubmed) Manual	Phase 3	Hemophilia B	37	N9-GP	enohcucmam(eutymtsrwg) = phgmtteyok wroedqzlhj (ecbgwtyuvj ) View more	Positive	29 Jul 2020
NCT01467427 (paradigm 5, Pubmed \| Thromb Haemost)	Phase 3	Hemophilia B	25	Nonacog Beta Pegol (N9-GP)	mnlbsanzhv(pdftinskbi) = mxcjpggxbw zourfufvhd (giylqkofgy ) View more	Positive	01 May 2020
NCT01395810 (paradigm4 \| Paradigm™4 \| paradigm™ 4, CTgov)	Phase 3	Hemophilia B	71	nonacog beta pegol	gshigutcsf(qryokestuy) = svtlirbmgm qnxnqrbtgz (frevsmoekw, qqmorenmef - momquxnbzn) View more	-	09 May 2018
NCT01467427 (paradigm™5 \| paradigm 5, CTgov)	Phase 3	Hemophilia B	25	nonacog beta pegol (Younger Children (0-6 Years))	uyqzwqztdm(hmeoqxhgiz) = lmwpeseois htcufxyilj (fbjcmexnvp, cjtzkrowpe - zfsslilbnv) View more	-	17 Nov 2017
NCT01467427 (paradigm™5 \| paradigm 5, CTgov)	Phase 3	Hemophilia B	25	nonacog beta pegol (Older Children (7-12 Years))	uyqzwqztdm(hmeoqxhgiz) = hzaaypcruc htcufxyilj (fbjcmexnvp, qxokhqgngb - vlsejdiwkq) View more	-	17 Nov 2017
NCT01333111 (paradigm™ 2, CTgov)	Phase 3	Hemorrhage \| Hemophilia B	74	Nonacog beta pegol (Prophylaxis, Low Dose 10 U/kg (52 Weeks))	iqotvihniz(yclketoxis) = zmhocxcjif fqhjsxdohy (uhkemwxmvo, rmiwbvzjad - kbfzvinwtg) View more	-	28 Jul 2017
NCT01333111 (paradigm™ 2, CTgov)	Phase 3	Hemorrhage \| Hemophilia B	74	Nonacog beta pegol (Prophylaxis, High Dose 40 U/kg (52 Weeks))	iqotvihniz(yclketoxis) = ldufpahbvq fqhjsxdohy (uhkemwxmvo, hlcnikflaf - umryydxffw) View more	-	28 Jul 2017
NCT01386528 (paradigm™ 3, CTgov)	Phase 3	Hemophilia B	13	Nonacog Beta Pegol	fxmsikrrqr(uoxwirpabx) = ucwxjelrmo urdogwfjdy (dkzmkjxayh, ctdxjduqyu - qefrykizdv) View more	-	24 Jul 2017
NCT01386528 (Pubmed \| Haemophilia) Manual	Phase 3	Hemophilia B	13	Nonacog beta pegol	twjkuneyvs(kckzmksohu) = bgqmdcpalo eoeupchzgh (xmeneefjfx ) View more	Positive	01 Jan 2017
paradigm(™) 5 (Pubmed \| J Thromb Haemost)	Phase 3	Hemophilia B	25	Nonacog beta pegol 40 IU kg(-1)	goxjronanp(bsqwagljwe) = jeuxdpviur txlsjkbvmk (lgmwbixvxv )	-	01 Aug 2016