Miglustat
Clinical data | |
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Trade names | Zavesca, Brazaves, Opfolda |
Other names | OGT 918, 1,5-(butylimino)-1,5-dideoxy-D-glucitol, N-butyl-deoxynojirimycin |
AHFS/Drugs.com | Monograph |
MedlinePlus | a604015 |
License data | |
Pregnancy category |
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Routes of administration | By mouth |
ATC code | |
Legal status | |
Legal status | |
Pharmacokinetic data | |
Bioavailability | 97% |
Protein binding | Nil |
Metabolism | Nil |
Elimination half-life | 6–7 hours |
Excretion | Kidney, unchanged |
Identifiers | |
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Miglustat, sold under the brand name Zavesca among others, is a medication used to treat type I
It was approved for medical use in the European Union in November 2002,[7][11] and for medical use in the United States in July 2003.[12][13]
Medical uses
Miglustat is
In the European Union, miglustat (Opfolda), in combination with cipaglucosidase alfa, is a long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α‑glucosidase [GAA] deficiency).[10]
Contraindications
Miglustat is contraindicated for people with neurological conditions, kidney problems, women who are pregnant, and men and women planning to conceive a child.[15]
Adverse effects
Serious side effects include pain, burning, numbness or tingling in the hands, arms, legs, or feet; shaking hands that cannot be controlled; changes in vision; and easy bruising or bleeding. Common side effects include gastrointestinal effects (including diarrhea, stomach pain or bloating, gas, loss of appetite, weight loss, upset stomach, vomiting, constipation), dry mouth, muscular effects (including weakness, muscle cramps, especially in the legs, feeling of heaviness in the arms or legs, unsteadiness when walking), back pain, dizziness, nervousness, headache, memory problems, and difficult or irregular menstruation (period).[15]
Mechanism of action
Type I Gaucher's disease is an
Earlier treatments on the market (
Chemistry
Miglustat is an iminosugar, a synthetic analogue of D-glucose[22] and a white to off-white crystalline solid that has a bitter taste.[23]
Society and culture
Legal status
Miglustat has been approved in the EU, Canada, and Japan for treating progressive neurological complications in people with Niemann–Pick disease, type C (NPC).[24][25][26][27][28]
On 26 April 2023, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Opfolda, intended for the treatment of glycogen storage disease type II (Pompe disease) in combination with cipaglucosidase alfa.[10] The applicant for this medicinal product is Amicus Therapeutics Europe Limited.[10] Opfolda is a hybrid medicine of Zavesca which has been authorized in the EU since 2002.[10] Opfolda contains the same active substance as Zavesca but in a lower strength.[10] It is also authorized for a different indication and can only be used in combination with cipaglucosidase alfa.[10] Miglustat (Opfolda) was approved for medical use in the European Union in June 2023.[8][29]
Research
In July 2004, Actelion started a clinical trial of miglustat to treat Tay–Sachs disease, particularly late-onset Tay–Sachs with an estimated enrollment of 10 subjects; the trial ended August 2007.[30]
In November 2007, Actelion initiated a clinical trial with miglustat in people with
N-butyldeoxynojirimycin interferes with the secretion of
References
- ^ "Miglustat (Zavesca) Use During Pregnancy". Drugs.com. 4 February 2020. Archived from the original on 28 November 2020. Retrieved 7 August 2020.
- ^ "Summary for ARTG Entry:122957 Zavesca miglustat 100 mg capsules blister pack". Therapeutic Goods Administration.[permanent dead link]
- ^ "Zavesca (miglustat) 100 mg hard capsules - Summary of Product Characteristics (SmPC)". (emc). Archived from the original on 25 February 2021. Retrieved 7 August 2020.
- ^ "Zavesca- miglustat capsule". DailyMed. 20 April 2023. Retrieved 2 October 2023.
- ^ "Yargesa- miglustat capsule". DailyMed. 12 July 2023. Retrieved 2 October 2023.
- ^ https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215211s000lbl.pdf
- ^ a b c "Zavesca EPAR". European Medicines Agency (EMA). 17 September 2018. Archived from the original on 5 August 2020. Retrieved 7 August 2020.
- ^ a b "Opfolda EPAR". European Medicines Agency (EMA). 7 July 2023. Retrieved 7 July 2023.
- ^ "Yargesa EPAR". European Medicines Agency. 11 April 2023. Retrieved 2 October 2023.
- ^ a b c d e f g "Opfolda: Pending EC decision". European Medicines Agency. 26 April 2023. Archived from the original on 27 April 2023. Retrieved 27 April 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ^ European Medicines Agency. Human Medicines Database. Zavesca (miglustat) Archived 7 January 2016 at the Wayback Machine Page Accessed 1 September 2014.
- ^ "Drug Approval Package: Zavesca (Miglustat) NDA #021348". U.S. Food and Drug Administration (FDA). 4 April 2002. Archived from the original on 27 February 2021. Retrieved 9 August 2020.
- ^ Actelion Press Release August 2003. Zavesca approved -- first oral treatment option for type 1 Gaucher disease Archived 3 March 2016 at the Wayback Machine
- S2CID 6681399.
- ^ a b American Society of Health-System Pharmacists, Inc. for the Public Library of Medicine. Miglustat on MedlinePlus Archived 5 July 2016 at the Wayback Machine Accessed 1 September 2014
- PMID 23233555.
- S2CID 72975032.
- PMID 22563238.
- ^ "Shire Announces FDA Approval Of VPRIV(TM) (velaglucerase Alfa For Injection) For The Treatment Of Type I Gaucher Disease". Medicalnewstoday.com. Archived from the original on 13 June 2011. Retrieved 13 August 2012.
- ^ Yukhananov A (1 May 2012). "U.S. FDA approves Pfizer/Protalix drug for Gaucher". Chicago Tribune. Reuters. Retrieved 2 May 2012.[permanent dead link]
- ^ Actelion. FDA Advisory Briefing Book for Miglustat (Ogt 918, Zavesca) in Niemann-Pick Type C Disease NDA 021-348/S-007 Archived 9 May 2017 at the Wayback Machine Prepared for the Endocrinologic and Metabolic Drugs Advisory Committee meeting, 1 December 2009
- PMID 21988669.
- ^ European Medicines Agency 1 April 2003 Scientific discussion related to approval of Zavesca Archived 24 September 2015 at the Wayback Machine.
- ^ UK Medicines Information. New Drugs Online Report for miglustat Archived 4 March 2016 at the Wayback Machine
- ^ Staff, The Pharma Letter. 4 April 2012. Actelion drops setipiprant, gets miglustat approval in Japan Archived 31 October 2018 at the Wayback Machine
- ^ Kevin Grogan for PharmaTimes. 10 March 2010. FDA rejects Actelion's Zavesca for rare NP-C disease Archived 3 September 2014 at the Wayback Machine
- ^ Actelion Press Release. 23 March 2010 Zavesca (Miglustat) First Treatment Available in Canada for Rare Progressive Niemann-Pick Type C Disease Archived 17 May 2017 at the Wayback Machine
- PMID 32897301.
- ^ "Opfolda Product information". Union Register of medicinal products. 27 June 2023. Archived from the original on 28 June 2023. Retrieved 3 July 2023.
- ^ Clinical trial number NCT00672022 for "Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses" at ClinicalTrials.gov
- ^ Clinical trial number NCT00537602 for "OGT 918 in the Treatment of Cystic Fibrosis" at ClinicalTrials.gov
- PMID 22281182.
- PMID 8134380.
- PMID 8794361.
- PMID 18753929.