RegulationBreakthrough Therapy (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Fast Track (United States)

Structure/Sequence

Molecular FormulaC24H30FN3O7S

InChIKeySQXUKOJKIWCALK-AAXLQGCPSA-N

CAS Registry1629063-78-0

Clinical Trials associated with Venglustat

NCT05718258

/ CompletedPhase 1

A Phase 1, Multi-center, Parallel, Open-label, Pharmacokinetic, Safety, and Tolerability Study of Venglustat Given as a Single Dose in Adult Participants With Mild, Moderate, and Severe Hepatic Impairment and in Matched Participants With Normal Hepatic Function

This is a parallel, Phase 1, four arm, open-label, single dose, multicenter study to evaluate the impact of hepatic impairment on venglustat exposure following treatment with venglustat.
The purpose of this study is to assess the effect of mild, moderate, and severe hepatic impairment on PK, safety, and tolerability of venglustat compared with normal hepatic function in male and female participants aged 18 to 79 years.
Study details include:
* The total study duration per participant will be up to 42 days, including up to 21 days for screening and approximately 21 days from institutionalization to the end of study (EOS).
* Institutionalization is mandatory until the activities on D5 have been completed.
* Each participant will receive a single dose of venglustat.
* For hepatically impaired participants there will be a screening visit, a multi-day institutionalization visit, and 7 site visits after D5 discharge, including the end of study (EOS) visit.
* For healthy volunteers there will be a screening visit, a multi-day institutionalization visit and 3 site visits after D5 discharge, including the end of study (EOS) visit.

Start Date18 Jan 2023

Sponsor / Collaborator

Sanofi

NCT05280548

/ Active, not recruitingPhase 3

A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy

This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy.
* Study visits will take place approximately every 3 to 6 months
* Participants who complete the randomized period may continue to the long-term extension (LTE) to receive venglustat for up to additional 45 months with the total study duration up to 5.3 years maximum.

Start Date03 May 2022

Sponsor / Collaborator

Sanofi

NCT05222906

/ Active, not recruitingPhase 3

A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (GD3) Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ERT)

This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions every two weeks for improvement or stabilization of the neurological manifestations and maintenance of systemic disease stability in participants aged ≥12 and <18 years and adult patients with Gaucher disease Type 3 (GD3) who have been treated with Enzyme Replacement Therapy (ERT) for at least 3 years.

Start Date18 Apr 2022

Sponsor / Collaborator

Sanofi

100 Clinical Results associated with Venglustat

100 Translational Medicine associated with Venglustat

100 Patents (Medical) associated with Venglustat

Literatures (Medical) associated with Venglustat

01 Jan 2026·GENETICS IN MEDICINE

Venglustat in GM2 gangliosidoses and related disorders: Results of the AMETHIST randomized controlled and basket trials

Article

Author: Thibault, Nathan ; Goyeau, Harmonie ; Jones, Simon A ; Zheng, Riliang ; Minini, Pascal ; Cox, Timothy M ; Nakashima, Ichiro ; O'Callaghan, Mar ; Batsu, Isabela ; Hahn, Andreas ; Tifft, Cynthia J ; Kowthalam, Madhurima Uppara ; Giugliani, Roberto ; Perlman, Susan

PURPOSE:

To evaluate efficacy and safety of venglustat for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) and cognate diseases.

METHODS:

The AMETHIST phase 3, randomized, double-blind, placebo-controlled study evaluated oral venglustat (N = 40) vs placebo (N = 19) in adults with late-onset GM2 gangliosidoses. Coprimary endpoints were annual percent change on the 9-Hole Peg Test and percent change in cerebrospinal fluid (CSF) GM2 ganglioside from baseline to week 104. A secondary population of participants with cognate diseases (N = 16) received open-label venglustat in a "basket" trial.

RESULTS:

CSF GM2 decreased by 47.6% (90% CI: -52.6, -42.6) with venglustat versus 11.3% (90% CI: -18.3, -4.4) with placebo (difference: -36.2 [90% CI: -44.8, -27.7], P < .0001). The annual percent change in 9-Hole Peg Test was 2.49% (90% CI: 0.28, 4.74) with venglustat versus 0.95% (90% CI: -2.16, 4.15) with placebo (difference: 1.54% [90% CI: -2.33, 5.39], P = .74). Decreased CSF GM2 concentrations did not correlate with clinical endpoints. Secondary population participants remained clinically stable. The most common adverse events were fall, headache, and contusion with placebo and fall, and COVID-19 and headache with venglustat.

CONCLUSION:

In adults with late-onset GM2 gangliosidoses, oral venglustat decreased CSF GM2 concentrations but without clinical improvement in the endpoints assessed. No new safety findings were observed.

01 Aug 2025·BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Drug–drug interaction study with itraconazole supplemented with physiologically based pharmacokinetic modelling to characterize the effect of CYP3A inhibitors on venglustat pharmacokinetics

Article

Author: Sharma, Jyoti ; Li, Li ; Crawford, Nigel ; Li, Yi ; Sahi, Jasminder ; Zhang, Yan‐Yan ; Macha, Sreeraj ; Cartot‐Cotton, Sylvaine

Aims:

Venglustat is an oral glucosylceramide synthase inhibitor under clinical investigation to treat various lysosomal storage diseases. Metabolism is a main pathway for its elimination in humans with CYP3A being the major contributor. This study aims to evaluate effect of CYP3A inhibition (using itraconazole) on venglustat exposure and to develop and validate a physiologically based pharmacokinetic (PBPK) model to assess effects of additional CYP3A inhibitors of varying potencies on venglustat pharmacokinetics.

Methods:

An open‐label, single‐sequence, 2‐period drug–drug interaction (DDI) study was conducted in healthy subjects with coadministration of multiple twice daily oral doses of 100 mg itraconazole against a single dose of 15 mg venglustat. A minimal PBPK model was developed using available physicochemical, in vitro and in vivo pharmacokinetic data and validated using data from relevant venglustat clinical studies including the itraconazole DDI study. Effects of additional CYP3A inhibitors on venglustat exposure were predicted.

Results:

Coadministration with itraconazole increased venglustat area under the concentration–time curve by 2.03‐fold (90% confidence interval [90%CI]: 1.81–2.27). Venglustat steady‐state area under the concentration–time curve during a dosing interval following coadministration with strong (clarithromycin), moderate (fluconazole) and weak (fluvoxamine and cimetidine; with CYP2D6 inhibition turned off) CYP3A inhibitors is predicted to increase by 1.74‐ (5th–95th centile, 1.30–2.49), 1.52‐ (1.23–1.88), 1.08‐ (1.03–1.15) and 1.08‐fold (1.04–1.12), respectively.

Conclusion:

The effect of itraconazole on venglustat exposure was quantified clinically, and a minimal PBPK model was successfully developed, validated and applied to assess DDI effect of additional CYP3A inhibitors on venglustat. The results help to further understand the DDI potential with venglustat and will inform dose recommendations with comedications.

23 Jan 2025·Nature

Photochemical permutation of thiazoles, isothiazoles and other azoles

Article

Author: Xu, Yan ; Yildiz, Dilara Berna ; Alonso, Maialen ; dos Santos, Thiago ; Bossart, Martin ; Buettner, Cornelia S. ; Llaveria, Josep ; Lonardi, Giovanni ; Buettner, Cornelia S ; Dos Santos, Thiago ; Roure, Baptiste ; Derdau, Volker ; Leonori, Daniele ; Ruffoni, Alessandro ; Méndez, María

Thiazoles and isothiazoles are privileged motifs in drug and agrochemical discovery^1,2. The synthesis of these derivatives is generally approached, designed and developed on a case-by-case basis. Sometimes, the lack of robust synthesis methods to a given target can pose considerable difficulties or even thwart the preparation of specific derivatives for further study^3,4. Here we report a conceptually different approach in which photochemical irradiation can be used to alter the structure of thiazoles and isothiazoles in a selective and predictable manner. On photoexcitation, these derivatives populate their π,π* singlet excited states that undergo a series of structural rearrangements, leading to an overall permutation of the cyclic system and its substituents. This means that once the initial heteroaromatic scaffold has been prepared, it can then function as an entry point to access other molecules by selective structural permutation. This approach operates under mild photochemical conditions that tolerate many chemically distinct functionalities. Preliminary findings also show the potential for extending this method to other azole systems, including benzo[d]isothiazole, indazole, pyrazole and isoxazole. This strategy establishes photochemical permutation as a powerful and convenient method for the preparation of complex and difficult-to-access derivatives from more available structural isomers.

News (Medical) associated with Venglustat

18 Mar 2026

·www.biospace.com

Press Release: Sanofi’s venglustat earns Breakthrough Therapy designation in the US for type 3 Gaucher disease

Sanofi’s venglusta t earns Breakthrough Therapy designation in the US for type 3 Gaucher disease Paris, March 18, 2026 . The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to venglustat, a novel, investigational oral glucosylceramide synthase inhibitor (GCSi), for the treatment of neurological manifestations of type 3 Gaucher disease (GD3), a rare lysosomal storage disorder. The designation is based on data from the LEAP2MONO phase 3 study (clinical study identifier: NCT05222906 ) in which patients receiving venglustat demonstrated statistically significant improvements in neurological symptoms measured by a global test score that included assessments for ataxia (mSARA) and cognition (RBANS) compared with patients receiving the enzyme replacement therapy (ERT), imiglucerase (p=0.007). In the study, venglustat was well tolerated overall with no new safety signals compared with previous studies. The most commonly reported adverse events were headache (14.3% in the venglustat arm versus 18.2% in the ERT arm), nausea (14.3% versus 4.5%), spleen enlargement (14.3% versus 0), and diarrhea (14.3% versus 0). Gaucher disease (GD) is a rare inherited lysosomal storage disorder that results from a deficiency of an enzyme called glucocerebrosidase, leading to the accumulation of sugar-and-fat molecules called glycosphingolipids (GSL) in the spleen, liver, bone marrow, and lungs. There are three major forms of the disease: GD1, characterized by lack of central nervous system (CNS) involvement; GD2, marked by rapid neurological decline and severe neurocognitive symptoms; and GD3, marked by slower and more variable progression and symptom severity. In people with GD3, accumulation of GSLs in the CNS can result in neurological symptoms, in addition to the systemic manifestations seen in GD1, such as liver and spleen enlargement, anaemia, low platelet counts, and bone disease. Systemic manifestations of GD3 are treated with ERT but there are no approved treatments for the neurological symptoms. Venglustat works by reducing the abnormal accumulation of GSLs. It is designed to cross the blood-brain barrier to target the underlying pathology causing the neurological effects of GD3. “This regulatory milestone recognizes the significant unmet medical need for people living with type 3 Gaucher disease, particularly those experiencing progressive neurological deterioration,” said Karin Knobe , Global Head of Clinical Development, Rare Diseases at Sanofi. “The positive LEAP2MONO findings are an encouraging step forward in the research and development process, and we will continue collaborating with the FDA to advance this potential treatment option.” Venglustat was previously granted fast-track designation from the FDA for its potential use in GD3 as well as orphan designation for GD3 in the US, EU and Japan. Sanofi will pursue global regulatory filings for venglustat in GD3 during 2026. FDA Breakthrough Therapy designation is designed to expedite the development and review of medicines in the US that target serious or life-threatening conditions. Medicines qualifying for this designation must show preliminary clinical evidence that the drug may demonstrate substantial improvement on clinically significant endpoints over available medicines. About the LEAP2MONO study The LEAP2MONO phase 3 study was a double-blind, double-dummy, active-comparator, two-arm study that evaluated the efficacy and safety of once daily oral venglustat versus intravenous ERT every two weeks in adults and pediatric patients aged 12 and older with GD3. Forty-three patients were randomized [1:1] to receive venglustat and placebo infusion or ERT and placebo tablet. Patients must have been treated with ERT for at least three years and achieved therapeutic goals for systemic disease manifestations. The primary endpoints for the study were change in SARA modified total score and change in RBANS total scale index score for patients receiving venglustat versus those receiving ERT from baseline to week 52. Systemic key secondary endpoints include percent change in spleen volume, liver volume and platelet count and change in hemoglobin levels. Biomarker key secondary endpoints include percent change in cerebrospinal fluid and plasma GL1 and lyso-GL1. The LEAP2MONO study is ongoing and results from its open-label phase will be presented in the future when available. About venglustat Venglustat is a novel, investigational oral glucosylceramide synthase inhibitor (GCSi), designed to cross the blood-brain barrier (i.e., brain-penetrant), that has the potential to slow the progression of GD3 by inhibiting the abnormal GSL accumulation and its physio-pathologic consequences. GSLs are cellular building blocks whose abnormal accumulation is implicated in several rare diseases leading to both cell dysfunction and disease progression. About Sanofi Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Media Relations Sandrine Guendoul | +33 6 25 09 14 25 | sandrine.guendoul@sanofi.com Evan Berland | +1 215 432 0234 | evan.berland@sanofi.com Léo Le Bourhis | +33 6 75 06 43 81 | leo.lebourhis@sanofi.com Victor Rouault | +1 617 356 4751 | victor.rouault@sanofi.com Timothy Gilbert | +1 516 521 2929 | timothy.gilbert@sanofi.com Léa Ubaldi | +33 6 30 19 66 46 | lea.ubaldi@sanofi.com Ekaterina Pesheva | +1 410 926 6780 | ekaterina.pescheva@sanofi.com Investor Relations Thomas Kudsk Larsen |+ 44 7545 513 693 | thomas.larsen@sanofi.com Alizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.com Keita Browne | + 1 781 249 1766 | keita.browne@sanofi.com Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com Nina Goworek | +1 908 569 7086 | nina.goworek@sanofi.com Thibaud Châtelet | + 33 6 80 80 89 90 | thibaud.chatelet@sanofi.com Yun Li | +33 6 84 00 90 72 | yun.li3@sanofi.com Sanofi Forward Looking Statement This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future events and economic performance. Words such as “expect,” “anticipate,” “believe,” “intend,” “estimate,” “plan,” “can,” “contemplate,” “could,” “is designed to,” “may,” “might,” “potential,” “objective,” "attempt," “target,” “project,” "strategy," "strive," "desire," “predict,” “forecast,” “ambition,” “guideline,” "seek," “should,” “will,” "goal," or the negative of these, and similar expressions are intended to identify forward-looking statements. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the U.S Food and Drug Administration or the European Medicines Agency, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates; the fact that product candidates if approved may not be commercially successful; unexpected regulatory actions or delays, or government regulation generally; authorities’ decisions regarding whether and when to approve a product candidate; political pressure in the United States to mandate lower drug prices including “most favored nation” pricing for State Medicaid programs; the future approval and commercial success of therapeutic alternatives; Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general; risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation; trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the French Markest Authority (AMF) made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2025 or contained in our periodic reports on Form 6-K. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. In light of these risks, uncertainties and assumptions, you should not place undue reliance on any forward-looking statements contained herein. All trademarks mentioned in this press release are the property of the Sanofi group. Attachment Press Release

Clinical ResultPhase 3Fast TrackBreakthrough TherapyOrphan Drug

18 Mar 2026

·www.drugs.com

Sanofi’s Venglustat Earns Breakthrough Therapy Designation in the US for Type 3 Gaucher Disease

Paris, March 18, 2026 . The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to venglustat, a novel, investigational oral glucosylceramide synthase inhibitor (GCSi), for the treatment of neurological manifestations of type 3 Gaucher disease (GD3), a rare lysosomal storage disorder. The designation is based on data from the LEAP2MONO phase 3 study (clinical study identifier: NCT05222906) in which patients receiving venglustat demonstrated statistically significant improvements in neurological symptoms measured by a global test score that included assessments for ataxia (mSARA) and cognition (RBANS) compared with patients receiving the enzyme replacement therapy (ERT), imiglucerase (p=0.007). In the study, venglustat was well tolerated overall with no new safety signals compared with previous studies. The most commonly reported adverse events were headache (14.3% in the venglustat arm versus 18.2% in the ERT arm), nausea (14.3% versus 4.5%), spleen enlargement (14.3% versus 0), and diarrhea (14.3% versus 0). Gaucher disease (GD) is a rare inherited lysosomal storage disorder that results from a deficiency of an enzyme called glucocerebrosidase, leading to the accumulation of sugar-and-fat molecules called glycosphingolipids (GSL) in the spleen, liver, bone marrow, and lungs. There are three major forms of the disease: GD1, characterized by lack of central nervous system (CNS) involvement; GD2, marked by rapid neurological decline and severe neurocognitive symptoms; and GD3, marked by slower and more variable progression and symptom severity. In people with GD3, accumulation of GSLs in the CNS can result in neurological symptoms, in addition to the systemic manifestations seen in GD1, such as liver and spleen enlargement, anaemia, low platelet counts, and bone disease. Systemic manifestations of GD3 are treated with ERT but there are no approved treatments for the neurological symptoms. Venglustat works by reducing the abnormal accumulation of GSLs. It is designed to cross the blood-brain barrier to target the underlying pathology causing the neurological effects of GD3. “This regulatory milestone recognizes the significant unmet medical need for people living with type 3 Gaucher disease, particularly those experiencing progressive neurological deterioration,” said Karin Knobe , Global Head of Clinical Development, Rare Diseases at Sanofi . “The positive LEAP2MONO findings are an encouraging step forward in the research and development process, and we will continue collaborating with the FDA to advance this potential treatment option.” Venglustat was previously granted fast-track designation from the FDA for its potential use in GD3 as well as orphan designation for GD3 in the US, EU and Japan. Sanofi will pursue global regulatory filings for venglustat in GD3 during 2026. FDA Breakthrough Therapy designation is designed to expedite the development and review of medicines in the US that target serious or life-threatening conditions. Medicines qualifying for this designation must show preliminary clinical evidence that the drug may demonstrate substantial improvement on clinically significant endpoints over available medicines. About the LEAP2MONO study The LEAP2MONO phase 3 study was a double-blind, double-dummy, active-comparator, two-arm study that evaluated the efficacy and safety of once daily oral venglustat versus intravenous ERT every two weeks in adults and pediatric patients aged 12 and older with GD3. Forty-three patients were randomized [1:1] to receive venglustat and placebo infusion or ERT and placebo tablet. Patients must have been treated with ERT for at least three years and achieved therapeutic goals for systemic disease manifestations. The primary endpoints for the study were change in SARA modified total score and change in RBANS total scale index score for patients receiving venglustat versus those receiving ERT from baseline to week 52. Systemic key secondary endpoints include percent change in spleen volume, liver volume and platelet count and change in hemoglobin levels. Biomarker key secondary endpoints include percent change in cerebrospinal fluid and plasma GL1 and lyso-GL1. The LEAP2MONO study is ongoing and results from its open-label phase will be presented in the future when available. About venglustat Venglustat is a novel, investigational oral glucosylceramide synthase inhibitor (GCSi), designed to cross the blood-brain barrier (i.e., brain-penetrant), that has the potential to slow the progression of GD3 by inhibiting the abnormal GSL accumulation and its physio-pathologic consequences. GSLs are cellular building blocks whose abnormal accumulation is implicated in several rare diseases leading to both cell dysfunction and disease progression. About Sanofi Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Sanofi Forward Looking Statement This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future events and economic performance. Words such as “expect,” “anticipate,” “believe,” “intend,” “estimate,” “plan,” “can,” “contemplate,” “could,” “is designed to,” “may,” “might,” “potential,” “objective,” "attempt," “target,” “project,” "strategy," "strive," "desire," “predict,” “forecast,” “ambition,” “guideline,” "seek," “should,” “will,” "goal," or the negative of these, and similar expressions are intended to identify forward-looking statements. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the U.S Food and Drug Administration or the European Medicines Agency, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates; the fact that product candidates if approved may not be commercially successful; unexpected regulatory actions or delays, or government regulation generally; authorities’ decisions regarding whether and when to approve a product candidate; political pressure in the United States to mandate lower drug prices including “most favored nation” pricing for State Medicaid programs; the future approval and commercial success of therapeutic alternatives; Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general; risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation; trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the French Markest Authority (AMF) made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2025 or contained in our periodic reports on Form 6-K. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. In light of these risks, uncertainties and assumptions, you should not place undue reliance on any forward-looking statements contained herein. All trademarks mentioned in this press release are the property of the Sanofi group. Source: Sanofi

Clinical ResultFast TrackPhase 3Orphan DrugBreakthrough Therapy

12 Feb 2026

·pmlive.com

Sanofi announces positive results for venglustat in type 3 Gaucher disease

Sanofi has announced positive results from its study of venglustat in in adults and children over 12 with neurological manifestations of type 3 Gaucher disease (GD3). Gaucher disease is a rare, inherited lysosomal storage disease caused by deficiency of the glucocerebrosidase enzyme. This leads to molecules known as glycosphingolipids (GSLs) building up, especially in macrophages of the spleen, liver, bone marrow and lungs. There are three main types of Gaucher disease. In GD3, neurological symptoms such as ataxia and cognitive issues are seen alongside the systemic manifestations that characterise GD1. Enzyme replacement therapy (ERT) can be used to treat systemic symptoms of GD3, but there are currently no approved treatments for its neurological symptoms. The LEAP2MONO study showed that venglustat improved both neurological and non-neurological outcomes. Improvements in neurological symptoms, which were the primary endpoint of the study, were measured at week 52. These scores were then compared with those of patients receiving ERT. Venglustat was found to be well tolerated by participants, and no new safety signals were observed. Sanofi plans to file global regulatory applications for venglustat in GD3. Results from the LEAP2MONO study will be presented at the 22nd annual WORLDSymposium. Houman Ashrafian, executive vice president and head of R&D at Sanofi, said: “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.” Sanofi is also studying a potential indication of venglustat in Fabry disease, which is another rare lysosomal storage disease. The phase 3 PERIDOT study in this indication did not meet its primary endpoint, but a second phase 3 study in Fabry disease, CARAT, is ongoing.

Phase 3Clinical Result

100 Deals associated with Venglustat

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KEGG	Wiki	ATC	Drug Bank
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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hypertrophy, Left Ventricular	Phase 3	United States	Sanofi	03 May 2022
Hypertrophy, Left Ventricular	Phase 3	China	Sanofi	03 May 2022
Hypertrophy, Left Ventricular	Phase 3	Japan	Sanofi	03 May 2022
Hypertrophy, Left Ventricular	Phase 3	Austria	Sanofi	03 May 2022
Hypertrophy, Left Ventricular	Phase 3	Canada	Sanofi	03 May 2022
Hypertrophy, Left Ventricular	Phase 3	Czechia	Sanofi	03 May 2022
Hypertrophy, Left Ventricular	Phase 3	Denmark	Sanofi	03 May 2022
Hypertrophy, Left Ventricular	Phase 3	France	Sanofi	03 May 2022
Hypertrophy, Left Ventricular	Phase 3	Germany	Sanofi	03 May 2022
Hypertrophy, Left Ventricular	Phase 3	Greece	Sanofi	03 May 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04221451 (AMETHIST, CTgov)	Phase 3	Sandhoff Disease \| Tay-Sachs Disease	75	placebo+venglustat (PAP: PP: Placebo)	aubtygknrj(dcxgtvkjdy) = cmrfcyspov zbiygtquty (aiahnyxiun, 4.23) View more	-	28 Jan 2026
				venglustat (PAP: PP: Venglustat)	aubtygknrj(dcxgtvkjdy) = wdeohiyccv zbiygtquty (aiahnyxiun, 3.04) View more
NCT03523728 (STAGED-PKD, CTgov)	Phase 2/3	Polycystic Kidney, Autosomal Dominant	478	Placebo (Stage 1- Placebo)	ahvwqojyqe(nymycjrfbp) = hgrbqpwqnn ajmlswzgfi (glpfsmhxjr, bowdzaeqhg - hsgdabbykq) View more	-	09 Nov 2022
				Venglustat (Stage 1- Venglustat 8 mg)	ahvwqojyqe(nymycjrfbp) = ggmpwmrxhy ajmlswzgfi (glpfsmhxjr, evccjxlzpk - zutnhyeboh) View more
NCT04705051 (STAGED-PKD-EXT, CTgov)	Phase 3	Polycystic Kidney, Autosomal Dominant	24	Venglustat	pjhetuxsdp = descmooajg owlmbtvfof (quugimuxpj, bktnqqldvc - fasxwexcyy) View more	-	06 Oct 2022
NCT04221451 (AMETHIST, SSIEM2022)	Phase 3	Gangliosidoses, GM2	75	Venglustat	jpoguwadxv(kanerwjeez) = ivginwaysg wsqqzmflgv (jpprzswfkk ) View more	-	30 Aug 2022
NCT02906020 (MOVES-PD, MDS2020)	Phase 2	Hypotension, Orthostatic GBA mutation	196	Venglustat	tziqgvtwvt(szlcksrgwn) = gerppkgget elrxianehn (gjjsjwetvl, 7 - 94) View more	Positive	12 Sep 2020
				Placebo	tltzewtfcd(dwxiaptulr) = srqlszkmut wvplqvevth (ggndfmvrhl )
NCT02489344 \| NCT02228460 (ERA2020)	Phase 2	Fabry Disease plasma GL-3 \| eGFR	11	Venglustat	uqpkvhvbkw(evpfuauvfy) = oyslcvhfvh fyjrycfyuf (uafmlrctwo )	Positive	06 Jun 2020
				Placebo	uqpkvhvbkw(evpfuauvfy) = wkkedwmozu fyjrycfyuf (uafmlrctwo )
NCT02906020 (MOVES-PD, EAN2020)	Phase 2	Parkinson Disease GBA mutations	29	Venglustat	zvpcojwval(tunudefubx) = shoxxivdha icbcxwfgjt (jgjyplfzwt )	Positive	22 May 2020
				Placebo	zvpcojwval(tunudefubx) = ckazukfdvq icbcxwfgjt (jgjyplfzwt )
NCT02228460 (CTgov)	Phase 2	Fabry Disease	11	GZ/SAR402671	xzzwcztksp(ydlgavsawh) = gaghqrhnqu khkhokrjdv (uhuxipjbro, vkkxuexwur - ijkdlxtlac) View more	-	17 Dec 2019
NCT02489344 (CTgov)	Phase 2	Fabry Disease	8	GZ/SAR402671	enjdbeqcvj = kxekhmghfp rpytqwrbpj (btbtgglfqp, tftxyetllh - rrmlzcrrpb) View more	-	09 Dec 2019
NCT02843035 (LEAP, SSIEM2019)	Phase 2	Gaucher Disease, Type 3 GL-1	5	Venglustat in combination with imiglucerase	cgckedbhqj(zqugxwqotj) = qsgwuobzpc zrmqlszqqr (ipzdmqvfbe, 36.8) View more	-	25 Aug 2019

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