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Drug

Miglustat

Approved · EMA
ClassCeramide glucosyltransferase inhibitorEmerging researchLate-stage development
Also known as N-Butylmoranoline, Opfolda, Yargesa, Zavesca+7 more

N-Butylmoranoline, Opfolda, Yargesa, Zavesca, Butyldeoxynojirimycin, N-(n-Butyl)deoxynojirimycin, N-butyl-1-deoxynojirimycin, N-butyl-deoxynojirimycin, n-Butyl deoxynojirimycin, 1,5-(butylimino)-1,5-dideoxy, D-glucitol, Miglustatum.

RxNorm402316ChEMBLCHEMBL1029ATCA16AX06UNIIADN3S497AZ
2
Research papers
2
Active clinical trials
Ceramide glucosyltransferase
Primary target
5
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease

Clinical trial2026-06-01Primary completion · ClinicalTrials.gov

An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years

Clinical trial2026-01-22Results expected Q3 2027 · ClinicalTrials.gov

A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo

Clinical trial2025-09-11Results posted · ClinicalTrials.gov

Survey of Miglustat Therapeutic Effects on Neurological and Systemic Symptoms of Infantile Type of Sandhoff and Taysachs Diseases

Clinical trial2025-09-10Terminated · ClinicalTrials.gov

Open-label, Non Comparative, Multi-center Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Oral Miglustat as a Maintenance Therapy After a Switch From Enzyme Replacement Therapy in Adult Patients With Stable Type 1 Gaucher Disease

Clinical trial2025-02-04Results posted · ClinicalTrials.gov

Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study.

Research2023-07-21Orphanet journal of rare diseases

Approval: Opfolda (EMA)

Regulatory2023-06-26EMA

A Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat (Zavesca) for the Treatment of Niemann Pick Type C Disease (NPC) in Chinese Subjects

Clinical trial2022-03-25Completed · ClinicalTrials.gov

Approval: Miglustat Dipharma (EMA)

Regulatory2019-02-18EMA

Approval: Miglustat Gen.Orph (EMA)

Regulatory2017-11-09EMA

Approval: Yargesa (EMA)

Regulatory2017-03-22EMA

Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study.

Research2012-12-27Orphanet journal of rare diseases

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Miglustat
Aliases & brands
N-ButylmoranolineOpfoldaYargesaZavescaButyldeoxynojirimycinN-(n-Butyl)deoxynojirimycinN-butyl-1-deoxynojirimycinN-butyl-deoxynojirimycinn-Butyl deoxynojirimycin1,5-(butylimino)-1,5-dideoxy, D-glucitolMiglustatum
RxNorm CUI
402316
ChEMBL ID
CHEMBL1029
ATC codes
A16AX06
UNII
ADN3S497AZ
Primary mechanism
Ceramide glucosyltransferase inhibitor
Regulatory jurisdictions
ema

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

Ceramide glucosyltransferase
UGCG · Q16739
INHIBITORCeramide glucosyltransferase inhibitor

Regulatory timeline

5 events

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2002-11-20
Latest approval
2023-06-26
Authorities
EMA
Total events
5
emaEuropean Medicines Agency· 5 events
2023-06-26Approval
Approval: Opfolda (EMA)
Indication: Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ?- glucosidase [GAA]… Show full indication

Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ?- glucosidase [GAA] deficiency).

Evidence ↗
2019-02-18Approval
Approval: Miglustat Dipharma (EMA)
Indication: Miglustat Dipharma is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat Dipharma may be used only in the treatment of… Show full indication

Miglustat Dipharma is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat Dipharma may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable. Miglustat Dipharma is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.

Evidence ↗
2017-11-09Approval
Approval: Miglustat Gen.Orph (EMA)
Indication: Miglustat Gen.Orph is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.&nbsp; Miglustat Gen.Orph may be used only in the treatment… Show full indication

Miglustat Gen.Orph is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.&nbsp; Miglustat Gen.Orph may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable. Miglustat Gen.Orph is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

20 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
20
registered trials across all phases
LATEST COMPLETION 2022
2
Active studies
1
Recruiting
11
Late-stage (III+)
14
Completed
4
Discontinued
PHASE DISTRIBUTIONn = 20
Phase 11Phase 28Phase 37Phase 43Phase N / A1

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat (Zavesca) for the Treatment of Niemann Pick Type C Disease (NPC) in Chinese Subjects

CompletedPH IVActelionn=172022

Synergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G)

TerminatedPH IVUniversity of Minnesotan=162019

Glycosphingolipid Inhibition and Spermatogenesis in Man: A Pilot Study

CompletedPH IVUniversity of Washingtonn=82006

An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years

RecruitingPH IIIAmicus Therapeuticsn=362027

An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease

Active, not recruitingPH IIIAmicus Therapeuticsn=212026

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat (Zavesca) for the Treatment of Niemann Pick Type C Disease (NPC) in Chinese Subjects

CompletedPH IVActelionn=172022

Phase 2 Pharmacological Trial to Evaluate the Safety of Miglustat Administration in Subjects With Spastic Paraplegia 11 (TreatSPG11)

CompletedPH IIIRCCS Fondazione Stella Marisn=102021

A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo

CompletedPH IIIAmicus Therapeuticsn=1252021

Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients Homozygous for the F508del Mutation? A Randomized, Double Blind Placebo-controlled Study.

CompletedPH IICliniques universitaires Saint-Luc- Université Catholique de Louvainn=102011

Application of Miglustat in Patients With Niemann-Pick Type C

CompletedPH IIINational Taiwan University Hospitaln=52010

Open-label, Non Comparative, Multi-center Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Oral Miglustat as a Maintenance Therapy After a Switch From Enzyme Replacement Therapy in Adult Patients With Stable Type 1 Gaucher Disease

CompletedPH IIIActelionn=422010

Research activity

2 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time
20122023
Major research themes3
Enzyme Therapy1Nervous System Diseases1Niemann-Pick Disease, Type C1
Journals, researchers & institutions
Top journals
  • Orphanet journal of rare diseases2
Leading researchers
  • Amato D1
  • Brassier A1
  • Broué P1
  • Cances C1
  • Chabrol B1
  • Cox TM1
  • Dahmani-Rabehi B1
  • Eyer D1
Leading institutions
free-text, unnormalised
  • Department of Clinical Biochemistry1
  • Haguenau Hospital1
  • INSERM U1037 (Cancer Research Centre of Toulouse)1
  • Jean Verdier University Hospital1
  • Kremlin-Bicêtre University Hospital1
  • La Timone University Hospital1

Related drugs

1 matches

Drugs sharing diseases, protein targets and literature with this one. Ranked by graph evidence (shared targets + diseases weighted above co-mentions).

1-Deoxynojirimycin

Studied across 2 of the same disease areas as Miglustat in the shared literature.

2 shared diseases1 shared paper
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.