Delving into the Latest Updates on Recombinant coagulation factor VIII ( Sino Cell Technologies) with Synapse

Overview

Basic Info

Drug Type

Recombinant coagulation factor

Synonyms

B-domain-deleted recombinant factor-VIII, BDDr FVIII, BDDr factor-VIII

+ [6]

Target

F10

Action

stimulants

Mechanism

F10 stimulants(Coagulation factor X stimulants)

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication

Hemophilia AHemorrhage

Inactive Indication-

Originator Organization

Sinocelltech Group Ltd.

Active Organization

Shenzhou Cell Engineering Co. Ltd.

Sinocelltech Group Ltd.

Inactive Organization-

Drug Highest PhaseApproved

First Approval Date

China (28 Jul 2021),

Hemophilia A

Regulation-

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Structure/Sequence

Sequence Code 1294233

Source: *****

Primary: To evaluate the safety of SCT800 for prophylaxis in untreated patients with hemophilia A. Secondary: To evaluate the effectiveness of SCT800 for prophylaxis in untreated patients with hemophilia A. To evaluate the safety and effectiveness of SCT800 for the treatment of bleeding episodes in untreated patients with hemophilia A.

Start Date26 Apr 2024

Sponsor / Collaborator

Shenzhou Cell Engineering Co. Ltd.

NCT05888870

/ RecruitingPhase 4IIT

The Outcome Comparison of Immune Tolerance Induction Therapy Using SCT800 Combined With Daratumumab or SCT800 Alone in Hemophilia A Adolescents and Adults With High Titer Inhibitor: a Non-randomized Controlled Trial

To evaluate the time of response, sustained remission rate, and relapse rate of CD38 monoclonal antibody (Daratumumab) combined with SCT800 (rFVIII) in the treatment of hemophilia A adolescents and adults with high titer inhibitors.

Start Date22 Nov 2023

Sponsor / Collaborator

Hematology Hospital of Chinese Academy of Medical Sciences

[+1]

CTR20191772

/ CompletedPhase 3

评估SCT800在既往接受过因子VIII治疗的重型甲型血友病患者（＜12岁）中预防治疗的有效性、安全性和药代动力学研究

[Translation] A study to evaluate the efficacy, safety and pharmacokinetics of SCT800 as a preventive treatment in patients with severe hemophilia A (<12 years old) who have previously received factor VIII therapy

评估SCT800在既往接受过因子VIII（FVIII）治疗的重型甲型血友病儿童患者（<12岁）中进行预防性治疗的有效性。

[Translation]

To evaluate the efficacy of SCT800 as a preventive treatment in pediatric patients (<12 years) with severe hemophilia A who have received prior factor VIII (FVIII) therapy.

Start Date28 May 2020

Sponsor / Collaborator

Shenzhou Cell Engineering Co. Ltd.

100 Clinical Results associated with Recombinant coagulation factor VIII ( Sino Cell Technologies)

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100 Translational Medicine associated with Recombinant coagulation factor VIII ( Sino Cell Technologies)

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100 Patents (Medical) associated with Recombinant coagulation factor VIII ( Sino Cell Technologies)

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845

Literatures (Medical) associated with Recombinant coagulation factor VIII ( Sino Cell Technologies)

25 Mar 2025·Blood Advances

Cost-effectiveness of Voncento prophylaxis vs on-demand treatment in von Willebrand disease in the United Kingdom

Article

Author: McDade, Cheryl ; Yan, Songkai ; Escolar, Ginés ; Castaman, Giancarlo ; Millar, Carolyn ; Miesbach, Wolfgang ; Thomas, Will ; Wilson, Michele ; Tomic, Radovan

Abstractvon Willebrand factor (VWF) concentrates may be required for on-demand treatment (ODT) or long-term prophylaxis (LTP) in von Willebrand disease (VWD). This study assesses the cost-effectiveness of LTP compared with ODT in patients with VWD treated with Voncento in the United Kingdom. A Markov structure was developed to estimate the quality-adjusted life years (QALYs) and costs of VWD treatment over a lifetime horizon. Treatment options included ODT or LTP. For both options, we assumed plasma-derived VWF/factor VIII 2.4:1 (Voncento) as the VWF product used. Clinical parameters were obtained from published literature and Voncento’s summary characteristics. Utility weights were obtained from published literature. Costs (in 2021 GBP [£]) and outcomes were discounted annually by 3.5%. Sensitivity analyses were conducted. Three baseline annual bleed rate (ABR) scenarios (11, 26.5, and 39.6) were considered. In the base-case analyses, Voncento LTP resulted in lower costs (–£831 206) and greater QALYs (6.14) vs ODT. Savings were primarily due to reductions in product use required (–£529 571) and bleed-related other medical costs (–£301 352). Compared with ODT, LTP also resulted in 322.52 fewer major bleeds and 515.68 fewer minor bleeds over a lifetime horizon. Probabilistic sensitivity analyses showed dominance in 96.12% of simulations and cost-effectiveness in 97.68% of simulations. For the 39.6 ABR scenario also, LTP was dominant compared with ODT. Results suggest that Voncento LTP is more effective and cost saving than ODT in the United Kingdom for patients with VWD with higher ABR. Prophylaxis for patients with frequent bleeds is likely to be a cost-saving and effective strategy.

01 Feb 2025·Journal of Thrombosis and Haemostasis

Comparing one stage, chromogenic assay results and discrepancies with bleeding phenotype and genetic variants in females with hemophilia A

Article

Author: Said, Mohammed ; Escobar, Miguel ; Menon, Neethu ; Rodriguez, Nidra ; Saroukhani, Sepideh ; Brown, Deborah ; Larson, Joanna ; Srivaths, Lakshmi

BACKGROUNDIncreasing evidence in females with hemophilia A has shown significant bleeding symptoms. Accurate factor (F) VIII activity (FVIII:C) measurement is essential to assign correct diagnosis and severity. Assay discrepancies reported in male patients with hemophilia A are not well studied in females.OBJECTIVESOur research sought to assess the association of FVIII:C levels by one-stage versus chromogenic assays and the assay discrepancy with bleeding phenotype and genetic mutation in females with hemophilia A.METHODSData from 64 females with hemophilia A from our center were reviewed with center's institutional review board approval. Descriptive statistics, chi-squared, Fisher's exact, and Bland-Altman plot analysis were applied.RESULTSAbnormal International Society on Thrombosis and Haemostasis bleeding assessment tool score was seen in 52% (FVIII:C <40 IU/dL: 72%/73%; FVIII:C ≥40 IU/dL: 41%/45%; by one-stage and chromogenic assays, respectively), more often in adults and postmenarchal patients. Assay discrepancies were present, direct more often than inverse. Based on one assay result only, 11 (17%) patients would have been incorrectly diagnosed and 1 (1.5%) patient would have been assigned incorrect hemophilia severity. No associations of genetic variants with FVIII:C levels and bleeding phenotype were found.CONCLUSIONOur study emphasizes the importance of evaluating female patients with hemophilia A for bleeding phenotype. Our study is the first to document prevalence of assay discrepancies exclusively in females with hemophilia A, which can lead to inaccurate diagnosis and severity assignment. Future larger, multicenter studies are needed to validate our findings, to help develop recommendations regarding the appropriate use of assays for accurate diagnosis and to further elucidate the association of genetic variants with bleeding phenotype and assay discrepancies.

01 Feb 2025·Journal of Thrombosis and Haemostasis

Intensive FVIII replacement in hemophilia patients with hypertrophic synovium: a randomized study

Article

Author: Calcaterra, Ilenia Lorenza ; Martinoli, Carlo ; Santoro, Rita Carlotta ; Marino, Renato ; Di Minno, Matteo Nicola Dario ; Baldacci, Erminia ; Valeri, Federica ; Pasta, Gianluigi

BACKGROUNDHypertrophic synovium (HS) is a marker of disease activity in persons with hemophilia (PwH). Although some recommendations suggest intensifying prophylaxis in PwH with HS, no validated schedules are available.OBJECTIVESWe explored the efficacy of intensive factor VIII (FVIII) replacement treatment in PwH with HS.METHODSIn a randomized, open-label study, PwH with HS were randomized to receive pharmacokinetics-driven prophylaxis targeting a FVIII trough level of 8% to 12% (intensive treatment arm [ITA]) or 3% to 5% (standard treatment arm [STA]). The primary outcome was HS reduction/resolution in the 2 treatment arms.RESULTSA total of 39 PwH were randomized to ITA and 36 to STA. During the study, we found a lower annual bleeding rate and a higher rate of annual bleeding rate zero in the ITA than in the STA. HS reduction/resolution was reported in 35.9% of cases in the ITA and 8.4% in the STA. In detail, in the ITA ,10.3% achieved HS reduction and 25.6% complete HS resolution, as compared to 5.6% and 2.8% in the STA. Cox regression showed that ITA was associated with HS reduction/resolution (hazard ratio: 4.75; 95% CI: 1.36-16.57; P = .014) and HS complete resolution (hazard ratio: 10.79; 95% CI: 1.38-84.45; P = .023). The analysis of the 127 joints with HS (54 elbows, 41 knees, and 32 ankles) consistently confirmed similar results.CONCLUSIONIn this randomized study, we found a ∼5-fold higher rate of HS reduction/resolution and a ∼10-fold higher rate of HS resolution in the ITA than in the STA.

100 Deals associated with Recombinant coagulation factor VIII ( Sino Cell Technologies)

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External Link

KEGG	Wiki	ATC	Drug Bank
-	-	B02BD02	-

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemophilia A	China	Sinocelltech Group Ltd.	28 Jul 2021

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hemorrhage	Phase 3	China	Shenzhou Cell Engineering Co. Ltd.	26 Apr 2024
Hemophilia A	Discovery	-	Sinocelltech Group Ltd.	20 Dec 2019

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03947320 (NEWS) Manual	Phase 3	Hemophilia A	68	SCT800 (0-5yr)	(wmclbipeen) = zppugkxvns bwsyzjgwwk (ntueklckze )	Positive	04 Jul 2022
				SCT800 (6-11yr)	(wmclbipeen) = ujljxopnxg bwsyzjgwwk (ntueklckze )
ISTH2021	Not Applicable	-	-	FVIII	mcotjplqes(rdflcpyuib) = cwablaigxr scmwsnudjv (igbxzkrbpd )	-	17 Jul 2021
				FVIII	mcotjplqes(rdflcpyuib) = kjhmtsiazp scmwsnudjv (igbxzkrbpd )
ISTH2020	Not Applicable	Headache FVIII	-	FVIII	butfhvkfxt(qxaoxjeduu) = ftoabgnpwa qwadtanifx (ohxliugdak )	-	12 Jul 2020
NCT03815318 (ISTH2020)	Phase 3	Hemophilia A	73	SCT800 25-50 IU/kg every other day	wnydhdqghu(djeuuumphd) = No patient developed inhibitors mhfhtqjpxi (xnmnbzpbjx ) View more	Positive	12 Jul 2020
				SCT800 25-50 IU/kg three times a week
EBMT2019	Not Applicable	Graft vs Host Disease Factor VIII \| von Willebrand Factor	70	FVIII (cGvHD patients)	hdhxjklsdt(kdzqzcrpyv) = xbcyaqtzgf acbotqvfrs (rxpftgeevj ) View more	-	24 Mar 2019
				FVIII (controls)	hdhxjklsdt(kdzqzcrpyv) = aaardcdenv acbotqvfrs (rxpftgeevj ) View more
ASGCT2018	Not Applicable	-	11	FVIII (Transplanted with mcoET3-LV transduced human PLCs)	sekmxpxogj(dsiwjopusf) = khyvfukmxl zbtwrartva (etilbhzqfm )	Positive	01 May 2018

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Biosimilar

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Recombinant coagulation factor VIII ( Sino Cell Technologies)

Overview

Basic Info

Structure/Sequence

Related

External Link

R&D Status

Clinical Result

Translational Medicine

Deal

Core Patent

Clinical Trial

Approval

Biosimilar

Regulation