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Miglustat | CAS No: 72599-27-0 | GMP-certified suppliers
A medication that treats mild to moderate type 1 Gaucher's disease and progressive neurological symptoms in Niemann-Pick disease type C when enzyme replacement therapy is unsuitable.
Therapeutic categories
Primary indications
- 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)
Product Snapshot
- Miglustat is an oral small molecule available in capsule and powder formulations
- It is primarily used for treating mild to moderate type 1 Gaucher's disease and neurological symptoms of Niemann-Pick disease type C
- The product holds regulatory approval in the US, EU, and Canada
Clinical Overview
Pharmacologically, miglustat is an N-alkylated imino sugar structurally related to D-glucose and classified within the piperidine chemical class. It functions as a competitive and reversible inhibitor of glucosylceramide synthase, the enzyme catalyzing the first step in glycosphingolipid synthesis, specifically the formation of glucosylceramide. In Gaucher disease, deficiency of glucocerebrosidase leads to accumulation of glucosylceramide in macrophage lysosomes, causing organomegaly, cytopenias, and bone complications. By inhibiting glucosylceramide synthase, miglustat reduces biosynthesis of glycosphingolipids, thereby lowering substrate load and enabling residual glucocerebrosidase activity to mitigate accumulation.
In NP-C, miglustat’s mechanism includes reduction of neurotoxic storage lipids such as gangliosides GM2 and GM3, lactosylceramide, and glucosylceramide, which may attenuate neuronal damage. Additional effects on intracellular calcium homeostasis related to sphingosine storage have been proposed to influence NP-C pathogenesis.
Pharmacokinetic characteristics include oral bioavailability, systemic distribution, and renal elimination. Safety profile considerations include gastrointestinal disturbances, potential tremors, and occasional weight loss; monitoring is recommended during treatment. Miglustat was initially developed as an anti-HIV agent but was not pursued for this indication due to safety and efficacy limitations at therapeutic doses.
Notable brand names include Zavesca. From an API sourcing perspective, ensuring high purity and robust control of synthetic impurities, along with comprehensive characterization conforming to pharmacopeial or regulatory standards, is crucial for consistent clinical performance. Manufacturers should maintain stringent quality assurance processes due to the critical role of miglustat in specialist therapeutic contexts.
Identification & chemistry
| Generic name | Miglustat |
|---|---|
| Molecule type | Small molecule |
| CAS | 72599-27-0 |
| UNII | ADN3S497AZ |
| DrugBank ID | DB00419 |
Pharmacology
| Summary | Miglustat is a competitive, reversible inhibitor of glucosylceramide synthase, reducing the biosynthesis of glycosphingolipids which accumulate in lysosomes due to enzyme deficiencies in disorders such as Gaucher's disease and Niemann-Pick type C. By lowering substrate levels, miglustat facilitates enhanced activity of residual glucocerebrosidase, decreasing lipid accumulation in macrophages and peripheral cells. Additionally, miglustat’s modulation of glycosphingolipid synthesis may affect intracellular calcium homeostasis, addressing pathogenic mechanisms underlying neurodegeneration in Niemann-Pick type C. |
|---|---|
| Mechanism of action | Miglustat functions as a competitive and reversible inhibitor of the enzyme glucosylceramide synthase, the initial enzyme in a series of reactions which results in the synthesis of most glycosphingolipids. The goal of treatment with miglustat is to reduce the rate of glycosphingolipid biosynthesis so that the amount of glycosphingolipid substrate is reduced to a level which allows the residual activity of the deficient glucocerebrosidase enzyme to be more effective (substrate reduction therapy), reducing the accumulation of glucocerebroside in macrophages. In vitro and in vivo studies have shown that miglustat can reduce the synthesis of glucosylceramide-based glycosphingolipids. In clinical trials, miglustat improved liver and spleen volume, as well as hemoglobin concentration and platelet count. Inhibition of glycosphingolipid synthesis has also shown to reduce intracellular lipid storage, improve fluid-phase endosomal uptake and normalize lipid transport in peripheral blood B lymphocytes of NP-C patients, which results in a decrease in the potentially neurotoxic accumulation of gnagliosides G<sub>M2</sub> and G<sub>M3</sub>, lactosylceramide and glucosylceramide, possibly preventing further neuronal damage. Other studies have also suggested that miglustat may indirectly modulate intracellular calcium homeostasis through its effects on glucosylceramide levels, and evidence has shown that an initiating factor in the pathogenesis of NP-C may be impaired calcium homeostasis related to sphingosine storage. Therefore, the effect that miglustat exerts on intracellular calcium levels may influence an important underlying pathogenic mechanism of NP-C. |
| Pharmacodynamics | Miglustat, an N-alkylated imino sugar, is a synthetic analogue of D-glucose. Miglustat is an inhibitor of the enzyme glucosylceramide synthase, which is a glucosyl transferase enzyme responsible for catalyzing the formation of glucosylceramide (glucocerebroside). Glucosylceramide is a substrate for the endogenous glucocerebrosidase, an enzyme that is deficient in Gaucher's disease. The accumulation of glucosylceramide due to the absence of glucocerebrosidase results in the storage of this material in the lysosomes of tissue macrophages, leading to widespread pathology due to infiltration of lipid-engorged macrophages in the viscera, lymph nodes, and bone marrow. This results in secondary hematologic consequences including sever anemia and thrombocytopenia, in addition to the characteristic progressive hepatosplenomegaly, as well as skeletal complications including osteonecrosis and osteopenia with secondary pathological fractures. |
Targets
| Target | Organism | Actions |
|---|---|---|
| Ceramide glucosyltransferase | Humans | inhibitor |
ADME / PK
| Absorption | Mean oral bioavailability is 97%. |
|---|---|
| Half-life | The effective half-life of miglustat is approximately 6 to 7 hours. |
| Protein binding | Miglustat does not bind to plasma proteins. |
| Metabolism | There is no evidence that miglustat is metabolized in humans. |
Formulation & handling
- Miglustat is a water-soluble small molecule intended for oral administration, typically formulated as capsules or powders.
- The piperidine structure and high solubility suggest good oral bioavailability without special formulation for lipophilicity enhancement.
- Administration timing should be consistent daily, but the compound can be taken with or without food, indicating minimal food interaction impact.
Regulatory status
| Lifecycle | The API has key patents in the United States that expired in 2013, with additional patents extending protection until 2033-2036. It is marketed in mature pharmaceutical markets including the US, EU, and Canada. |
|---|
| Markets | US, EU, Canada |
|---|
Supply Chain
| Supply chain summary | Miglustat is primarily manufactured and packaged by Actelion Pharmaceuticals, reflecting a single originator company involved in its production. Its branded products have a presence across multiple markets, including the US, EU, and Canada. Given the patent expirations ranging from 2013 to as late as 2036, the landscape includes both expired and active patent protections, indicating the potential for existing generic competition as well as continued exclusivity periods. |
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Safety
| Toxicity | Miglustat has been administered at doses of up to 3000 mg/day (approximately 10 times the recommended starting dose administered to Gaucher patients) for up to six months in Human Immunodeficiency Virus (HIV)-positive patients. Adverse events observed in the HIV studies included granulocytopenia, dizziness, and paresthesia. Leukopenia and neutropenia have also been observed in a similar group of patients receiving 800 mg/day or above. |
|---|
- High-dose exposure (≥800 mg/day) may induce hematological effects including leukopenia, neutropenia, and granulocytopenia
- Neurological adverse effects such as dizziness and paresthesia have been reported at elevated doses
- Careful handling required to minimize exposure due to potential myelosuppressive and neurotoxic properties
Miglustat is a type of Glycosides
Glycosides are a prominent subcategory of pharmaceutical active pharmaceutical ingredients (APIs) that play a crucial role in the development of various drugs and medicines. These compounds are characterized by the presence of a glycosidic bond, which connects a sugar molecule (glycone) to a non-sugar component (aglycone). Glycosides have gained significant attention in the pharmaceutical industry due to their diverse biological activities and therapeutic potential. They are widely used as natural sources for drug discovery and development. Many plants produce glycosides, and these natural compounds exhibit a wide range of pharmacological effects, such as anti-inflammatory, anticancer, antimicrobial, and antioxidant properties.
The extraction and isolation of glycosides from natural sources require specialized techniques like chromatography, solvent extraction, and purification methods. Once isolated, glycosides undergo rigorous testing and evaluation to determine their safety, efficacy, and dosage requirements.
Pharmaceutical companies utilize glycosides in the formulation of various medications, including cardiovascular drugs, anticancer agents, and anti-diabetic medications. Glycosides also serve as precursors for the synthesis of other pharmaceutical compounds, contributing to the development of new drugs and therapies.
As research in the field of glycosides continues to advance, scientists are exploring their potential applications in drug delivery systems, bioavailability enhancement, and targeted drug delivery. These innovative approaches aim to optimize the therapeutic benefits of glycosides and improve patient outcomes.
In conclusion, glycosides are a vital subcategory of pharmaceutical APIs, offering immense potential for drug development and therapeutic applications. Continued research and exploration of glycosides are crucial for expanding our understanding of their properties and unlocking their full pharmaceutical potential.
Miglustat (Glycosides), classified under Cardiac stimulants
Cardiac stimulants are a crucial category of pharmaceutical active pharmaceutical ingredients (APIs) used in the treatment of cardiac disorders. These medications are designed to enhance the functioning of the heart by stimulating its electrical impulses and increasing its contractility.
Cardiac stimulants work by targeting specific receptors in the heart, promoting the release of neurotransmitters such as norepinephrine and epinephrine. These neurotransmitters bind to adrenergic receptors, leading to an increased heart rate and force of contraction, which helps improve cardiac output.
One commonly used cardiac stimulant API is Dobutamine. Dobutamine acts primarily on beta-1 adrenergic receptors in the heart, increasing the strength of cardiac contractions while minimizing the impact on heart rate. This makes it a valuable medication in cases of acute heart failure or during cardiac stress testing.
Another well-known cardiac stimulant API is Isoproterenol. Isoproterenol acts on both beta-1 and beta-2 adrenergic receptors, resulting in increased heart rate, contractility, and relaxation of the smooth muscles in the bronchi. It is commonly used in the treatment of bradycardia, heart block, and certain types of asthma.
Cardiac stimulant APIs play a vital role in cardiovascular medicine and are often used in emergency situations or as temporary measures to improve heart function. However, it is important to note that their use requires careful monitoring and should be administered under medical supervision due to potential side effects such as increased blood pressure, arrhythmias, and myocardial ischemia.
In conclusion, cardiac stimulant APIs are a critical category of pharmaceutical ingredients used to enhance heart function. Medications like Dobutamine and Isoproterenol act on specific receptors in the heart, leading to increased contractility and heart rate. While these medications provide important therapeutic benefits, their use should be closely monitored by medical professionals due to potential side effects.
Miglustat API manufacturers & distributors
Compare qualified Miglustat API suppliers worldwide. We currently have 1 companies offering Miglustat API, with manufacturing taking place in 1 different countries. Use the table below to review supplier type, countries of origin, certifications, product portfolio and GMP audit availability.
| Supplier | Type | Country | Product origin | Certifications | Portfolio |
|---|---|---|---|---|---|
| Amino Chemicals | Producer | Malta | Malta | CoA, GMP | 20 products |
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