A Single Arm, Prospective, Open Label, Multicenter Study to Evaluate Efficacy and Safety of One-year Maximum Dosage in Chinese Label of Imiglucerase Treatment in Chinese Patients Who Are Diagnosed as Gaucher Disease Type Ⅲ

Primary Objective
* To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
* To evaluate the safety profile of imiglucerase in maximum dose in the label (60U/kg, IV biweekly) in Chinese patients.
Secondary Objective
* To evaluate the efficacy on viscera manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
* To evaluate the efficacy on bone disease of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
* To evaluate the effect on quality of life of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ

Start Date02 Mar 2021

Sponsor / Collaborator

Sanofi

NCT04787887

/ CompletedPhase 1

A Randomized, Double-blind, 2-treatment, 2-period, Crossover Phase I Study to Compare the PK, Safety and Tolerability of 60 IU/kg of Abcertin, and EU-sourced Cerezyme® in Healthy Volunteers Following a Single Intravenous Administration

Primary Objective:
To compare the pharmacokinetics of Abcertin to the reference product, EU-sourced Cerezyme, after single intravenous administration of 60 IU/kg.
Secondary Objective:
To compare the safety, tolerability and immunogenicity of Abcertin to the reference formulation, EU-sourced Cerezyme, after single intravenous administration of 60 IU/kg.

Start Date29 Jan 2020

Sponsor / Collaborator

ISU Abxis Co., Ltd.

NCT03485677

/ CompletedPhase 3

Open Label, Two Cohort (With and Without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients With Gaucher Disease Type 1 and Type 3

Primary Objective:
Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (≥2 to <18 years old).
Secondary Objective:
Evaluate the efficacy of eliglustat and quality of life in pediatric patients (≥2 to <18 years old).

Start Date11 Apr 2018

Sponsor / Collaborator

Sanofi

100 Clinical Results associated with Imiglucerase

100 Translational Medicine associated with Imiglucerase

100 Patents (Medical) associated with Imiglucerase

226

Literatures (Medical) associated with Imiglucerase

01 Dec 2025·GENE THERAPY

Systemically delivered lipid nanoparticle-mRNA encoding lysosomal acid β-glucosidase restores the enzyme deficiency in a murine Gaucher disease model

Article

Author: Wang, Shasha ; Wang, Zihao ; Guo, Zhijun ; Zhang, Zhang ; Chen, Yanni ; Liu, Yuanqing ; Quan, Xiaojiang

Gaucher disease (GD) is a rare genetically inherited illness caused by loss of lysosomal acid β-glucosidase (β-GCase) that leads to progressive accumulation of substrates, sphingolipid glucosylceramide (GL1) and glucosylsphingosine (lyso-GL1). The protein-based enzyme replacement therapy (ERT) requires frequent dosing due to short drug half-life causing challenges in long-term patient compliance. JCXH-301 is a lipid nanoparticle (LNP) encapsulated messenger RNA (mRNA) encoding β-GCase. Intravenous administration of JCXH-301 delivered the target mRNA to various tissues in mice with intracellular expression of β-GCase predominantly in macrophages and dendritic cells in the spleen and bone marrow. In GBA1 D427V homozygous mice treated with JCXH-301, the dose-dependent in vivo production of functional β-GCase resulted in reduction of serum lyso-GL1, a key biomarker of GD. The therapeutic effect of JCXH-301 was sustained for a duration significantly longer than that of protein-based ERT Cerezyme. JCXH-301 administration induced minimal pro-inflammatory cytokines in the liver and spleen. Taken together, these results provide proof-of-concept for using LNP-delivered mRNA as a new drug modality to restore the β-GCase genetic deficiency for GD treatment.

01 Dec 2025·Molecular Genetics and Metabolism Reports

Real-world experience of switching to taliglucerase among patients with Gaucher disease in Québec: A case series

Article

Author: Marie-Claude Miron ; François Mercier ; Angelo Rizzolo ; Nathalie Alos ; Jean-François Delisle ; Philippe M. Campeau ; Claude Meilleur

Enzyme replacement therapy (ERT) for Gaucher disease (GD) effectively prevents skeletal, visceral, and hematologic complications of this inherited, lysosomal storage disorder. Taliglucerase is one of the three commercially available ERT products and became the recommended first-line therapy in Québec, Canada in 2016. Thus, 19 patients were switched from imiglucerase to taliglucerase, but more than a quarter experienced significant side effects. Here, we summarize these patients' clinical course and describe 6 suspected product-related adverse-effects.

01 Jun 2025·Molecular Genetics & Genomic Medicine

A Randomized, Double‐Blind, 2‐Treatment, 2‐Period, Crossover Phase 1 Study to Compare the Pharmacokinetics, Safety and Tolerability of 60 IU/Kg of Abcertin and Cerezyme in Healthy Volunteers Following a Single Intravenous Administration

Article

Author: Kang, Eungu ; Wharton, Jessica ; Lee, Yongyoon ; Hwang, Soojin ; Lemech, Charlotte ; Kim, Dohyung ; Yoo, Han Wook ; Lee, Beom Hee

ABSTRACT:

Background:

Imiglucerase (Cerezyme; Sanofi, Paris, France), an analogue of β‐glucocerebrosidase produced by recombinant DNA technology, has been a safe and effective treatment for Gaucher disease (GD) for over 25 years. A new imiglucerase, Abcertin (Seongnam‐si, Gyeonggi‐do, Republic of Korea) has shown a similar safety and efficacy profile in previous clinical studies. This study compared the pharmacokinetics, immunogenicity, safety, and tolerability to EU‐sourced Cerezyme following a single 60 IU/kg dose.

Methods:

This phase 1, single‐center, randomized, double‐blind, two‐way crossover study enrolled 36 healthy volunteers aged 18–45 years. Participants were randomly assigned to receive either Abcertin or Cerezyme in a predetermined sequence.

Results:

Abcertin reached peak plasma concentrations at a median tmax of 61 min (range: 40–121 min). The mean Cmax, AUC0–last, and AUC0–inf were 115.4 mU/mL, 12,190 min·mU/mL, and 12,210 min mU/mL, respectively, indicating bioequivalence to Cerezyme. The mean t½, CL, and Vz were 6.88 min, 376.7 mL/min, and 3.62 L, respectively, and were comparable between the two treatments. One participant in the Cerezyme group developed anti‐drug antibodies, which were non‐neutralizing A total of 24 subjects experienced treatment‐emergent adverse event (TEAE). The most common TEAE was headache (3 in the Abcertin group and 5 in the Cerezyme group), followed by general disorders and administration site condition (3 in Abcertin group and 5 in Cerezyme group). Two participants in the Cerezyme sequence experienced severe TEAEs: one had a urinary tract infection, and the other developed urticaria, which leading to study withdrawal.

Conclusion:

Abcertin demonstrated pharmacokinetic equivalence to Cerezyme, with a comparable safety, immunogenicity, and tolerability profile. These findings support its potential as an affordable biosimilar for GD treatment.

News (Medical) associated with Imiglucerase

02 Feb 2026

·www.fiercebiotech.com

Sanofi\'s troubled GCS inhibitor fails phase 3 Fabry trial but scores in Gaucher disease

Venglustat was once considered as a potential \"pipeline in a pill.\"\n Sanofi’s pipeline features a number of once-hyped candidates that are struggling to deliver in the clinic, and the latest mixed phase 3 data from venglustat continue this trend.The French Big Pharma was studying venglustat, an oral, brain-penetrant glucosylceramide synthase (GCS) inhibitor, in a pair of late-stage studies of rare lysosomal storage disorders. One of these trials, called Peridot, assessed the pill in 122 patients with Fabry disease, while the Leap2Mono trial focused on 43 patients with type 3 Gaucher disease (GD3).The Peridot study missed its primary endpoint of demonstrating superiority in patient-reported improvement of symptoms—namely neuropathic pain in upper and lower extremities, as well as abdominal pain—compared to a placebo cohort. Sanofi explained this failure by pointing to a “reduction in neuropathic and abdominal pain … observed in both study arms.”It was better news in the Leap2Mono study, where the 21 patients who received venglustat saw statistically significant improvements in neurological symptoms at Week 52 as measured by scales for ataxia and for neuropsychological status. The improvement between the venglustat cohort and the 22 patients who received enzyme replacement therapy (ERT) worked out as a p-value of 0.007, Sanofi explained in its Feb. 2 release.When it came to non-neurological secondary endpoints like changes in spleen volume, liver volume and hemoglobin level, venglustat “performed as well as ERT,” according to the pharma.Venglustat was well tolerated, Sanofi said. The most common adverse events were headache, nausea, spleen enlargement and diarrhea—although reports of headache were higher in the ERT cohort.Based on the Leap2Mono data, Sanofi said it will seek regulatory approval for venglustat in GD3. The company already markets the ERT Cerezyme and the oral glucosylceramide synthase inhibitor Cerdelga for the disease.The failure of the Peridot study doesn’t mark the end of Sanofi’s Fabry ambitions, either. As well as its approved Fabry ERT Fabrazyme, Sanofi is still running a phase 3 study of venglustat on left cardiac ventricular mass index in patients with the disease.“These findings underscore Sanofi\'s commitment to rare disease research and the promise we aim to deliver for people living with these conditions,” Sanofi’s head of R&D Houman Ashrafian, Ph.D., said in this morning’s release.“What excites us most is the potential to address critical unmet medical needs,” Ashrafian added. “A daily pill could make a serious difference for Gaucher patients facing neurological challenges.”This morning’s phase 3 readouts continued a run of mixed data for venglustat, which had at one point been hailed as one of Sanofi\'s top prospects and a potential “pipeline in a pill” contender. Those ambitions were blunted as far back as 2021, when venglustat failed midstage studies in both Parkinson’s disease and autosomal dominant polycystic kidney disease. By 2024, venglustat’s options were narrowed down to just Fabry disease and GD3 after Sanofi gave up on GM2 gangliosidosis as an indication “based on the absence of positive trends on clinical endpoints.”While Sanofi remains hopeful of getting venglustat approved for GD3, the Fabry failure this morning also continues a longer-term trend of the drugmaker’s pipeline struggling to achieve its potential. The company’s R&D chief admitted to Fierce last year that clinical setbacks for its anti-OX40L-ligand antibody amlitelimab, its oral TNF inhibitor balinatunfib and its Regeneron-partnered IL-33 candidate itepekimab had resulted in a “mixed” 2025.On an earnings call with journalists last week, Sanofi CEO Paul Hudson directly acknowledged that it had been a “bumpy ride” for R&D.“I\'m well aware of the headlines—we had some challenges,” the CEO said at the time.

$Sanofi\'s troubled GCS inhibitor fails phase 3 Fabry trial but scores in Gaucher disease$

Phase 3Clinical ResultDrug Approval

02 Feb 2026

·firstwordpharma.com

Sanofi posts a win for venglustat in Gaucher study, but a miss in Fabry disease

Sanofi announced Monday mixed results from a pair of pivotal studies of venglustat in two rare lysosomal storage disorders, with the oral glucosylceramide synthase inhibitor (GCSi) recording a hit in type 3 Gaucher disease (GD3) and a miss in Fabry disease. The contrasting outcomes are the latest to emerge from the company's late-stage pipeline, following differing results earlier this year from the investigational OX40-ligand-blocking monoclonal antibody amlitelimab in two atopic dermatitis trials.The Phase III LEAP2MONO study randomised 43 adult and paediatric patients with neurological manifestations of GD3 to receive venglustat once-daily or enzyme replacement therapy (ERT). The trial's primary endpoints were change in Scale for Assessment and Rating of Ataxia (SARA) modified total score and change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score, with key secondary goals including percent change in spleen volume, liver volume and platelet count, as well as change in haemoglobin levels.Results showed that venglustat demonstrated statistically significant improvements in neurological symptoms as measured by both SARA and RBANS at week 52 compared to ERT. Sanofi added that the therapy also achieved three out of the four key secondary endpoints, performing as well as ERT on non-neurological outcomes, including changes in spleen volume, liver volume and haemoglobin levels.Regulatory filings in GD3"What excites us most is the potential to address critical unmet medical needs. A daily pill could make a serious difference for Gaucher patients facing neurological challenges," said Houman Ashrafian, head of R&D at Sanofi. The company indicated that based on the results, it will pursue global regulatory filings for venglustat in GD3.Sanofi currently markets the injectable therapy Cerezyme (imiglucerase) for Gaucher disease, as well as the oral drug Cerdelga (eliglustat) for those with type 1 disease. The drugmaker's portfolio also includes the injectable therapy Fabrazyme (agalsidase beta) for Fabry disease. However, the latest results dent its hopes of adding an oral Fabry disease therapy to its arsenal.Fabry setbackThe Phase III PERIDOT study randomised 122 patients aged 16 years and older with Fabry disease to receive venglustat or placebo. Sanofi noted that the trial failed to hit its primary endpoint of percent change from baseline on patient-defined most bothersome symptoms, as assessed by the Fabry Disease Patient-Reported Outcome (FD-PRO) instrument.According to the company, reductions in neuropathic and abdominal pain were observed in both study arms. Additional analyses of the data are ongoing, while the late-stage CARAT trial evaluating the effect of venglustat on left cardiac ventricular mass index in men and women with Fabry disease is ongoing.Findings from both LEAP2MONO and PERIDOT are scheduled to be presented this week at the annual WORLDSymposium.Aside from the positive results in GD3, Venglustat, which works by reducing the abnormal accumulation of sugar-and-fat molecules in cells and organs, has racked up a number of study failures. In 2024, Sanofi disclosed that the Phase III AMETHIST trial for GM2 gangliosidosis was discontinued based on “the absence of positive trends” for clinical endpoints, adding to a pivotal Phase II/III study setback in autosomal dominant polycystic kidney disease and another in Parkinson's disease with GBA mutations.

Phase 3Clinical Result

02 Feb 2026

·www.biopharmadive.com

Sanofi drug, inherited from Genzyme, goes 1-for-2 in rare disease trials

An experimental rare disease drug from Sanofi succeeded against one so-called lysosomal storage disorder but failed against another, the French pharmaceutical company said Monday.According to Sanofi, the drug, dubbed venglustat, missed its primary objective in a Phase 3 study testing it against Fabry disease. However, in another study in a form of Gaucher disease, the drug met its main goal and three out of four key secondary endpoints. Sanofi didnt provide details theyll be shared at medical meeting this week but said it intends to submit the Gaucher results to global regulatory authorities.Sanofi has viewed venglustat as a key part of its future for more than a decade. The company inherited the medication as part of an acquisition of the rare disease drug specialist Genzyme and, since then, has tested it against a variety of conditions. Its meant to lower the production of a kind of toxic substrate, in turn reducing the buildup of potentially dangerous materials in cells such as a certain fatty substance in Gaucher disease.The drugs development has been marred by clinical setbacks, however. Prior to Mondays announcement, venglustat failed studies in Parkinsons disease, a rare kidney condition, as well as another lysosomal storage disorder known as GM2 gangliosidosis.On Monday, Fabry was added to the list. In a Phase 3 trial, venglustat wasnt associated with a meaningful improvement in patient-reported pain scores, compared to a placebo. Sanofi said additional analyses are ongoing and more information will be shared at a future medical meeting. A second Phase 3 study of venglustat in Fabry disease is ongoing, Sanofi said.The drug did, though, succeed in a study of people with the Type 3 form of Gaucher, which affects brain function and can lead to progressive neurological impairment. While people with Gaucher can get an enzyme replacement therapy Sanofi sells one called Cerezyme those treatments dont help with the diseases effects on the brain.Sanofi tested venglustat against an enzyme replacement therapy in the study. It found that, after a year of treatment, those who received its therapy had statistically significant improvements, comparatively, on two tests evaluating neurological symptoms. The drug was well tolerated, though instances of nausea, diarrhea and spleen enlargement were higher among recipients, Sanofi said.“What excites us most is the potential to address critical unmet medical needs, said Houman Ashrafian, Sanofis head of research and development, in the statement. A daily pill could make a serious difference for Gaucher patients facing neurological challenges.The venglustat readouts come after a few recent pipeline setbacks for Sanofi. An eczema drug thats currently one of the companys top prospects produced results that fell short of expectations in a late stage trial. An important multiple sclerosis drug also has an uncertain future. '

Phase 3AcquisitionClinical Result

100 Deals associated with Imiglucerase

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KEGG	Wiki	ATC	Drug Bank
D03020	Imiglucerase	A16AB02	DB00053

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Gaucher Disease	China	Genzyme Corp.	26 Nov 2008
Gaucher Disease, Type 3	European Union	Sanofi	17 Nov 1997
Gaucher Disease, Type 3	Iceland	Sanofi	17 Nov 1997
Gaucher Disease, Type 3	Liechtenstein	Sanofi	17 Nov 1997
Gaucher Disease, Type 3	Norway	Sanofi	17 Nov 1997
Anemia	United States	Genzyme Corp.	23 May 1994
Bone Diseases	United States	Genzyme Corp.	23 May 1994
Gaucher Disease, Type 1	United States	Genzyme Corp.	23 May 1994
Hepatomegaly	United States	Genzyme Corp.	23 May 1994
Myeloid Splenomegaly	United States	Genzyme Corp.	23 May 1994
Thrombocytopenia	United States	Genzyme Corp.	23 May 1994

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT00364858 (CTgov)	Phase 4	Gaucher Disease, Type 1	95	Cerezyme (Q2 Cerezyme)	vbigrdwwkz = bnbsslmwlp jojsmfofla (bhpgrskzbr, vagavpfqaz - ecdmouvrla) View more	-	02 Sep 2009
				Cerezyme (Q4 Cerezyme)	vbigrdwwkz = vzqjrpmksx jojsmfofla (bhpgrskzbr, jaqmyxxskw - vposkcqnrl) View more

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