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

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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, Inc.Novo Nordisk Pharmaceuticals Pty Ltd.

+ [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·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. ; Rosa, C. ; Baques, A. ; Sueldo, E. ; Zirpoli, M. ; Ceresetto, J. ; Arias, M.

AbstractIntroductionHaemophilia 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.MethodsIt 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.ResultsThe 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.ConclusionAPTT 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 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: Holst, Helle ; Nielsen, Pernille Højlund ; Mrakotsky, Christine ; Shapiro, Kevin ; Walsh, Karin S ; Chan, Anthony K C ; Carcao, Manuel

BackgroundNonacog 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.ObjectivesTo 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.MethodsPTPs (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.ResultsOverall, 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.ConclusionThese 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 Jan 2024·Haemophilia

Indirect treatment comparisons of the gene therapy etranacogene dezaparvovec versus extended half‐life factor IX therapies for severe or moderately severe haemophilia B

Article

Author: Klamroth, Robert ; Bonner, Ashley ; Yan, Songkai ; Wang, Di ; Zhang, Xiang ; Walsh, Sarah ; Szafranski, Kirk ; Gomez, Keith ; Monahan, Paul E

AbstractIntroductionEtranacogene dezaparvovec gene therapy for haemophilia B demonstrated superior efficacy at 24 months in reducing bleeds versus a ≥6‐month lead‐in period of prophylaxis with FIX products in the phase 3 trial, HOPE‐B. In the absence of head‐to‐head comparisons of etranacogene dezaparvovec versus FIX products, indirect treatment comparisons (ITC) can be used.AimTo compare the efficacy of etranacogene dezaparvovec versus rIX‐FP, rFIXFc and N9‐GP using ITC, and support HOPE‐B results.MethodsData were leveraged from Phase 3 pivotal trials: HOPE‐B, PROLONG‐9FP, B‐LONG and Paradigm 2. Annualised bleeding rates (ABR), spontaneous (AsBR) and joint (AjBR) bleeding rates, percentage of patients with no bleeds, and FIX consumption were assessed using inverse probability of treatment weighting and matching adjusted indirect comparisons.ResultsEtranacogene dezaparvovec demonstrated statistically significantly lower bleeding rates versus all comparators. Rate ratios for ABR, AsBR and AjBR versus rIX‐FP were 0.19 (p < .0001), 0.08 (p < .0001) and 0.09 (p < .0001), respectively. Rate ratios for ABR, AsBR and AjBR versus rFIXFc were 0.14 (p < .0001), 0.13 (p = .0083) and 0.15 (p = .0111), respectively. Rate ratios for ABR and AsBR, versus N9‐GP were 0.24 (p = .0231) and 0.13 (p = .0071), respectively. Etranacogene dezaparvovec demonstrated significantly higher percentage of patients with no bleeds versus rIX‐FP and rFIXFc; odds ratios: 17.60 (p < .0001) and 5.65 (p = .0037), respectively. Etranacogene dezaparvovec resulted in significantly lower FIX consumption than all comparators.ConclusionsITC suggests that etranacogene dezaparvovec offers patients with haemophilia B (≤2% of normal FIX expression) a single dose treatment that can significantly reduce bleeding rates and eliminate routine infusions associated with FIX therapies.

1

News (Medical) associated with Nonacog beta pegol

22 Mar 2021

·www.globenewswire.com

89bio Presents Additional Analysis of Phase 1b/2a NASH Study at the Annual ENDO 2021 Conference

SAN FRANCISCO, March 20, 2021 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardio-metabolic diseases, today announced additional positive data from its Phase 1b/2a study of BIO89-100, a long-acting glycoPEGylated FGF21 analog, in patients with nonalcoholic steatohepatitis (NASH). The data will be presented in an on-demand poster presentation at ENDO 2021, the Endocrine Society’s annual meeting taking place virtually from March 20-23, 2021. “Excess liver fat is an important driver of disease progression for people living with NASH and can be associated with increased risk for cardiovascular events and even death,” said Hank Mansbach, chief medical officer of 89bio. “We are encouraged by new analyses from our Phase 1b/2a study that show BIO89-100 demonstrated clinically meaningful reductions in both liver fat volume and liver volume overall across all dosing groups. The data continues to highlight the promising clinical profile of BIO89-100 and supports further development of BIO89-100 in NASH and also severe hypertriglyceridemia.” New analyses of the Phase 1b/2a study data showed BIO89-100 treatment resulted in significant reductions in liver volume of up to 15% and liver fat volume of up to 65% in treated patients at 13 weeks compared to baseline, as measured by magnetic resonance imaging-proton density fat fraction (MRI-PDFF). These data extend the previously reported MRI-PDFF data in which BIO89-100 treatment resulted in up to 70% relative reduction in liver fat fraction relative to placebo treatment. Additionally, BIO89-100, as previously reported, demonstrated a favorable safety and tolerability profile, with rates of gastrointestinal side effects such as nausea, diarrhea and vomiting similar to placebo. The poster presentation details are as follows: Session: P02 - Integrated Physiology of Obesity and Metabolic Disease Poster Presentation: #53 Poster Title: BIO89-100 Demonstrated Robust Reductions in Liver Fat and Liver Fat Volume (LFV) by MRI-PDFF, Favorable Tolerability and Potential for Weekly (QW) or Every 2 Weeks (Q2W) Dosing in a Phase 1b/2a Placebo-Controlled, Double-Blind, Multiple Ascending Dose Study in NASH Presenting Author: Juan Pablo Frias, M.D. A copy of the poster presentation is also available for download via the 89bio website. About NASH NASH is the most advanced stage of nonalcoholic fatty liver disease (NAFLD). It is a complex metabolic disorder that causes fat buildup in the liver, as well as inflammation and eventually fibrosis, and it can worsen to cirrhosis and liver failure. NASH affects more than 16 million adults in the United States, and by 2030 its prevalence is predicted to increase by 63 percent. The exact cause of NASH is unknown, but it is commonly found in people with obesity and type 2 diabetes. While there are currently no approved treatments, the biopharmaceutical industry is actively involved in addressing this unmet medical need. About the Phase 1b/2a Study This clinical study was a multicenter, randomized, double-blind, placebo-controlled, multiple ascending dose-ranging trial. It was designed to assess the safety, tolerability, and PK properties of BIO89-100 as well as change in liver fat measured by MRI-PDFF and key biomarker assessments in patients with biopsy-proven NASH with fibrosis or patients with phenotypical NASH (PNASH). PNASH was defined as patients with steatosis greater than 10% who have central obesity and Type 2 diabetes or central obesity and evidence of liver injury. Both populations that were enrolled had similar disease characteristics at baseline. A total of 81 patients were randomized to receive weekly or every two weeks subcutaneous dosing of BIO89-100 or placebo for up to 12 weeks. Results observed across all dose groups from the trial add to a growing body of evidence demonstrating the promise of BIO89-100 for the treatment of NASH. Results showed robust reductions in liver fat and key liver markers. A strong efficacy profile and favorable tolerability were observed with weekly and every two-week dosing. About BIO89-100 BIO89-100 is a glycoPEGylated analog of FGF21 being developed for the treatment of NASH. 89bio has optimally engineered BIO89-100 using a proprietary glycoPEGylation technology to balance efficacy and longer dosing interval. Recent Phase 1b/2a data show BIO89-100 demonstrated a favorable safety and tolerability profile and robust reductions in liver fat and key lipid markers when dosed weekly (QW) or once every two weeks (Q2W). BIO89-100 is also being developed for the treatment of severe hypertriglyceridemia (SHTG) and is currently in a Phase 2 trial. About 89bio 89bio is a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardio-metabolic diseases. The company’s lead product candidate, BIO89-100, is a specifically engineered glycoPEGylated analog of FGF21. BIO89-100 is being developed for the treatment of nonalcoholic steatohepatitis (NASH) and severe hypertriglyceridemia (SHTG). 89bio is headquartered in San Francisco with operations in Herzliya, Israel. Investor Contact: Ryan Martins Chief Financial Officer investors@89bio.com Media Contact: Peter Duckler 773-343-3069 pduckler@w2ogroup.com

AntibodyCollaborate

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
Hemophilia B	Phase 3	MY	Novo Nordisk A/S	27 Apr 2011
Hemophilia B	Phase 3	TH	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	US	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	TH	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	MY	Novo Nordisk A/S	27 Apr 2011
Hemorrhage	Phase 3	FR	Novo Nordisk A/S	27 Apr 2011
Hemophilia B	Discovery	ZA	Novo Nordisk A/S	27 Apr 2011
Hemophilia B	Discovery	MK	Novo Nordisk A/S	27 Apr 2011
Hemophilia B	Discovery	TR	Novo Nordisk A/S	27 Apr 2011
Hemophilia B	Discovery	RU	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)	dkentbsemt(xfdsywvcle) = dwrbczccpl giorgszydw (atcmchhrnw, jimppaviha - uezvupavik) View more	-	29 Dec 2023
				nonacog beta pegol (Prophylaxis)	dkentbsemt(xfdsywvcle) = qybpgzldzr giorgszydw (atcmchhrnw, sjuzxfrstd - gozfpwsfgi) View more
NCT01467427 (paradigm 5, Pubmed \| Thromb Haemost)	Phase 3	Hemophilia B	25	Nonacog Beta Pegol (N9-GP)	vzwxzcecdq(dlxibamrrn) = trjabrfwpl jpesldrmjh (uvsodxyhxa ) View more	Positive	01 May 2020
NCT01395810 (paradigm4 \| Paradigm™4 \| paradigm™ 4, CTgov)	Phase 3	Hemophilia B	71	nonacog beta pegol	htlrtbirdv(mderdmgidv) = tmwlwtkdbs ubxzzgxzwz (gfohhdtvyx, wftammfook - nazjxqqgdp) View more	-	09 May 2018
NCT01467427 (paradigm™5 \| paradigm 5, CTgov)	Phase 3	Hemophilia B	25	nonacog beta pegol (Younger Children (0-6 Years))	bgirabaawv(sxjsfignql) = lropyuxqqh vyfhzzbfkd (rwhardwevp, ycmuuhsqjr - fnlqigebjy) View more	-	17 Nov 2017
				nonacog beta pegol (Older Children (7-12 Years))	bgirabaawv(sxjsfignql) = zezvoyxeuj vyfhzzbfkd (rwhardwevp, tkuintjgie - nwhifdwvwy) View more
NCT01333111 (paradigm™ 2, CTgov)	Phase 3	Hemorrhage \| Hemophilia B	74	Nonacog beta pegol (Prophylaxis, Low Dose 10 U/kg (52 Weeks))	awmwcocrpb(fjvqtjsivl) = zngkffdpop xifdlyakzd (vtelxydkpi, jytlsxylgs - sndjclqwxx) View more	-	28 Jul 2017
				Nonacog beta pegol (Prophylaxis, High Dose 40 U/kg (52 Weeks))	awmwcocrpb(fjvqtjsivl) = sdumprnebp xifdlyakzd (vtelxydkpi, sbauguhsbu - sshtupirpd) View more
NCT01386528 (paradigm™ 3, CTgov)	Phase 3	Hemophilia B	13	Nonacog Beta Pegol	wzpnrlytiv(eyrtvfdvlz) = mnwsiabpni fgqndczuic (mdxqmbylxh, vtjhqbrdsx - vhywpmlpww) View more	-	24 Jul 2017
NCT01386528 (Pubmed \| Haemophilia) Manual	Phase 3	Hemophilia B	13	Nonacog beta pegol	eqbjcwpeay(rrmlgpeekr) = npggeacgib srogdiaomt (hrmxfktlhg ) View more	Positive	01 Jan 2017
NCT01395810 (Paradigm™4, Pubmed \| J Heart Lung Transplant) Manual	Phase 3	Hemophilia B	71	Nonacog beta pegol (two once-weekly prophylaxis arms)	(dsmxhfmrns) = ceovrysyna pnuvcgmtqk (mpubtxhfwa, 0.00 - 2.23) View more	Positive	01 May 2016
				Nonacog beta pegol (an on-demand arm)	(dsmxhfmrns) = klfbppazrc pnuvcgmtqk (mpubtxhfwa, 0.00 - 2.03) View more