Miglustat

Overview

Miglustat, commonly marketed under the trade name Zavesca, is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids. It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. Miglustat is now the first and only approved therapy for patients with Niemann-Pick disease type C (NP-C). It has recently been approved for treatment of progressive neurological symptoms in adult and pediatric patients in the European Union, Brazil, and South Korea. Miglustat was first developed as an anti-HIV agent in the 1990s. However, clinical experience with miglustat showed that therapeutic levels of the drug could not be achieved in patients without a high incidence of adverse effect.

Indication

For the treatment of adult patients with mild to moderate type 1 (nonneuropathic) Gaucher's disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to constraints such as allergy, hypersensitivity, or poor venous access). Now approved in some countries for the treatment of progressive neurological symptoms in adult and pediatric patients with Niemann-Pick disease type C (NP-C).

Associated Conditions

Mild, moderate Gaucher Disease, Type 1

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
A Global Prospective Observational Registry of Patients With Pompe Disease	2023/11/07	NCT06121011	N/A	Recruiting	Amicus Therapeutics
An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of CLN3 Disease	2021/12/30	NCT05174039	Phase 1	Completed	Beyond Batten Disease Foundation
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18	2021/03/22	NCT04808505	Phase 3	Recruiting	Amicus Therapeutics
Testing Miglustat Administration in Subjects With Spastic Paraplegia 11	2021/02/24	NCT04768166	Phase 2	Completed	IRCCS Fondazione Stella Maris
Expanded Access for ATB200/AT2221 for the Treatment of IOPD	2020/03/31	NCT04327973	N/A	AVAILABLE	Amicus Therapeutics
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD	2019/10/24	NCT04138277	Phase 3	Completed	Amicus Therapeutics
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD	2019/04/11	NCT03911505	Phase 3	Recruiting	Amicus Therapeutics
Safety and Efficacy of Miglustat in Chinese NPC Patients	2019/04/10	NCT03910621	Phase 4	Completed	Actelion
Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease	2019/03/07	NCT03865836	N/A	AVAILABLE	Amicus Therapeutics
Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)	2019/01/30	NCT03822013	Phase 3	Recruiting	Tehran University of Medical Sciences

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
Miglustat	Zydus Pharmaceuticals USA Inc.	70710-2039	ORAL	100 mg in 1 1	3/17/2025
Zavesca	Actelion Pharmaceuticals US, Inc.	66215-201	ORAL	100 mg in 1 1	4/20/2023
MIGLUSTAT	ANI Pharmaceuticals, Inc.	43975-310	ORAL	100 mg in 1 1	8/31/2022
OPFOLDA	AMICUS THERAPEUTICS US, LLC	71904-300	ORAL	65 mg in 1 1	9/1/2023
Yargesa	Edenbridge Pharmaceuticals LLC.	42799-709	ORAL	100 mg in 1 1	10/11/2023
Miglustat	CoTherix, Inc.	10148-201	ORAL	100 mg in 1 1	4/25/2023

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Yargesa	Piramal Critical Care B.V.,Rouboslaan 32,2252 TR Voorschoten,Netehrlands	Authorised	3/22/2017
Yargesa	Piramal Critical Care B.V.,Rouboslaan 32,2252 TR Voorschoten,Netehrlands	Authorised	3/22/2017
Zavesca	Janssen Cilag International NV,Turnhoutseweg 30,B 2340 Beerse,Belgium	Authorised	11/20/2002
Miglustat Dipharma	Dipharma Arzneimittel GmbH,Offheimer Weg 33,65549 Limburg a. d. Lahn,Germany	Authorised	2/18/2019
Opfolda	Amicus Therapeutics Europe Limited,Block 1, Blanchardstown Corporate Park,Ballycoolin Road,Blanchardstown,Dublin 15,D15 AKK1,Ireland	Authorised	6/26/2023
Miglustat Gen.Orph	Gen.Orph,185 Bureaux de la Colline,92213 Saint Cloud Cedex,France	Authorised	11/9/2017

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
ZAVESCA miglustat 100 mg capsules blister pack	122957	Janssen-Cilag Pty Ltd	Medicine	A	10/23/2007
OPFOLDA miglustat 65 mg capsule bottle	438811	Amicus Therapeutics Pty Ltd	Medicine	A	12/16/2024

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
SANDOZ MIGLUSTAT	Sandoz Canada, Inc.	02456257	Capsule - Oral	100 MG	4/20/2020
ZAVESCA	Janssen Inc	02250519	Capsule - Oral	100 MG	5/26/2004
OPFOLDA	Amicus Therapeutics Canada Inc.	02556812	Capsule - Oral	65 MG	N/A

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
OPFOLDA 65 MG CAPSULAS DURAS	Amicus Therapeutics Europe Limited	1231737001	CÁPSULA DURA	Uso Hospitalario	Commercialized
YARGESA 100 MG CAPSULAS DURAS EFG	Piramal Critical Care B.V.	1171176001	CÁPSULA DURA	Uso Hospitalario	Commercialized
MIGLUSTAT DIPHARMA 100 MG CAPSULAS DURAS EFG	Dipharma Arzneimittel Gmbh	1181346002	CÁPSULA DURA	Uso Hospitalario	Commercialized
ZAVESCA 100 MG CAPSULAS DURAS	Janssen-Cilag International N.V	02238001	CÁPSULA DURA	Uso Hospitalario	Commercialized
MIGLUSTAT GEN.ORPH 100 MG CAPSULAS DURAS EFG	Gen.Orph S.A.S.	1171232001	CÁPSULA DURA	Uso Hospitalario	Commercialized

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

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The European Medicines Agency's Committee for Medicinal Products for Human Use has issued a positive opinion recommending approval of Aqneursa (levacetylleucine) for treating neurological manifestations of Niemann-Pick type C disease in adults and children.

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