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Eliglustat | CAS No: 491833-29-5 | GMP-certified suppliers
A medication that provides long-term treatment for type 1 Gaucher disease by reducing glucosylceramide accumulation in adult patients with specific CYP2D6 metabolizer phenotypes.
Therapeutic categories
Alimentary Tract and MetabolismCytochrome P-450 CYP2D6 InhibitorsCytochrome P-450 CYP2D6 Inhibitors (strength unknown)Cytochrome P-450 CYP2D6 SubstratesCytochrome P-450 CYP3A SubstratesCytochrome P-450 CYP3A4 Substrates
Generic name
Eliglustat
Molecule type
small molecule
CAS number
491833-29-5
DrugBank ID
DB09039
Approval status
Approved drug, Investigational drug
ATC code
A16AX10
Primary indications
- Eliglustat is a glucosylceramide synthase inhibitor indicated for the long-term treatment of type 1 Gaucher disease in adult patients who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test
- CYP2D6 ultra-rapid metabolizers may not achieve adequate eliglustat concentrations to achieve a therapeutic effect
- A specific dosage cannot be recommended for CYP2D6 indeterminate metabolizers
Product Snapshot
- Eliglustat is an oral small molecule formulated as coated capsules
- It is indicated for the treatment of type 1 Gaucher disease in adult patients with specific CYP2D6 metabolizer phenotypes
- Eliglustat is approved in key regulatory markets including the US, Canada, and the EU
Clinical Overview
Eliglustat (CAS number 491833-29-5) is an oral glucosylceramide synthase inhibitor approved for the long-term treatment of type 1 Gaucher disease in adult patients with specific CYP2D6 metabolizer phenotypes. Gaucher disease type 1 is a lysosomal storage disorder caused by deficiency of the enzyme acid β-glucosidase, leading to the accumulation of glucosylceramide in macrophage lysosomes. This accumulation results in the formation of Gaucher cells, which infiltrate organs such as the liver, spleen, and bone marrow, causing clinical manifestations including anemia, thrombocytopenia, and hepatosplenomegaly.
Eliglustat acts by selectively inhibiting glucosylceramide synthase, the rate-limiting enzyme catalyzing the synthesis of glucosylceramide, thereby reducing substrate accumulation. The specificity of eliglustat is evidenced by its lack of significant off-target inhibition on other glycosidases. Its mechanism provides a substrate reduction therapy alternative to enzyme replacement therapies like imiglucerase, velaglucerase alfa, and taliglucerase alfa.
Pharmacokinetic data indicate that eliglustat is primarily metabolized by the cytochrome P450 enzyme CYP2D6, with additional metabolism via CYP3A4. Due to this metabolic pathway, dosing is guided by the patient’s CYP2D6 genotype, as classified by FDA-cleared tests into extensive, intermediate, or poor metabolizer groups. These phenotypes determine dosage adjustments to achieve therapeutic plasma concentrations while avoiding toxicity. Ultra-rapid and indeterminate CYP2D6 metabolizers currently have no established dosing recommendations.
Eliglustat demonstrates a favorable safety profile at therapeutic doses; however, higher plasma concentrations can prolong cardiac conduction intervals (PR, QRS, QTcF), increasing the risk of arrhythmias. Consequently, eliglustat is contraindicated or requires caution in patients with preexisting cardiac conditions, long QT syndrome, or those taking interacting CYP2D6 or CYP3A4 inhibitors, as well as certain antiarrhythmic agents.
From a pharmaceutical sourcing perspective, eliglustat API procurement necessitates confirmation of chemical purity, stereochemical integrity, and impurity profiles consistent with regulatory standards. Given its classification as a benzo-1,4-dioxane derivative, verifying the absence of genotoxic impurities and consistent batch-to-batch quality is essential for compliance and clinical efficacy. Coordination with suppliers for appropriate documentation and validation of manufacturing processes is recommended to ensure supply chain reliability.
Eliglustat acts by selectively inhibiting glucosylceramide synthase, the rate-limiting enzyme catalyzing the synthesis of glucosylceramide, thereby reducing substrate accumulation. The specificity of eliglustat is evidenced by its lack of significant off-target inhibition on other glycosidases. Its mechanism provides a substrate reduction therapy alternative to enzyme replacement therapies like imiglucerase, velaglucerase alfa, and taliglucerase alfa.
Pharmacokinetic data indicate that eliglustat is primarily metabolized by the cytochrome P450 enzyme CYP2D6, with additional metabolism via CYP3A4. Due to this metabolic pathway, dosing is guided by the patient’s CYP2D6 genotype, as classified by FDA-cleared tests into extensive, intermediate, or poor metabolizer groups. These phenotypes determine dosage adjustments to achieve therapeutic plasma concentrations while avoiding toxicity. Ultra-rapid and indeterminate CYP2D6 metabolizers currently have no established dosing recommendations.
Eliglustat demonstrates a favorable safety profile at therapeutic doses; however, higher plasma concentrations can prolong cardiac conduction intervals (PR, QRS, QTcF), increasing the risk of arrhythmias. Consequently, eliglustat is contraindicated or requires caution in patients with preexisting cardiac conditions, long QT syndrome, or those taking interacting CYP2D6 or CYP3A4 inhibitors, as well as certain antiarrhythmic agents.
From a pharmaceutical sourcing perspective, eliglustat API procurement necessitates confirmation of chemical purity, stereochemical integrity, and impurity profiles consistent with regulatory standards. Given its classification as a benzo-1,4-dioxane derivative, verifying the absence of genotoxic impurities and consistent batch-to-batch quality is essential for compliance and clinical efficacy. Coordination with suppliers for appropriate documentation and validation of manufacturing processes is recommended to ensure supply chain reliability.
Identification & chemistry
| Generic name | Eliglustat |
|---|---|
| Molecule type | Small molecule |
| CAS | 491833-29-5 |
| UNII | DR40J4WA67 |
| DrugBank ID | DB09039 |
Pharmacology
| Summary | Eliglustat is a selective inhibitor of glucosylceramide synthase, reducing the synthesis of glucosylceramide and thereby decreasing its lysosomal accumulation in macrophages. It is primarily intended for long-term management of type 1 Gaucher disease by targeting the underlying glycosphingolipid biosynthesis pathway. Pharmacodynamically, eliglustat demonstrates high specificity with minimal off-target activity, influencing glycosphingolipid homeostasis through ceramide glucosyltransferase inhibition. |
|---|---|
| Mechanism of action | Eliglustat is a glucosylceramide synthase inhibitor used for the treatment of type 1 Gaucher disease. Gaucher disease is a rare genetic disorder characterized by the deficiency of acid β-glucosidase, an enzyme that converts glucosylceramide (also known as glucocerebroside) into glucose and ceramide. In patients with Gaucher disease, glucosylceramide is accumulated in the lysosomes of macrophages, leading to the formation of foam cells or Gaucher cells. Gaucher cells infiltrate the liver, spleen, bone marrow and other organs, leading to complications such as anemia, thrombocytopenia and hepatosplenomegaly. Eliglustat reduces the production of glucosylceramide by inhibiting glucosylceramide synthase, a rate-limiting enzyme in the production of glycosphingolipids. This lowers the amount of glucosylceramide that is available in lysosomes, and balances the deficiency of acid β-glucosidase. |
| Pharmacodynamics | Eliglustat is a specific inhibitor of glucosylceramide synthase (IC<sub>50</sub> =10 ng/mL). In vitro studies suggest that eliglustat has minimal or no off-target activity against other glycosidases, such as α-glucosidase I and II, and lysosomal and non-lysosomal glucosylceramidases. At 8 times the recommended dose (800 mg) and a mean peak concentration of 237 ng/mL, eliglustat did not have a clinically significant effect on QTc prolongation. However, modelling of PK/PD data predicts that at a plasma concentration of 500 ng/mL, PR, QRS and QTcF intervals increase 22, 7, and 13 msec, respectively. Since high plasma concentrations of eliglustat may increase the risk of cardiac arrhythmias, there are warnings and precautions for patients taking CYP2D6 or CYP3A4 inhibitors, those with specific CYP2D6 metabolizer status and different degrees of hepatic impairment. Depending on each case, the use of this drug is contraindicated, to be avoided, or requires dosage adjustment. Patients with preexisting cardiac disease (congestive heart failure, recent acute myocardial infarction, bradycardia, heart block, ventricular arrhythmia), long QT syndrome, or those taking Class IA or Class II antiarrhythmic drugs are advised to avoid eliglustat. |
Targets
| Target | Organism | Actions |
|---|---|---|
| Ceramide glucosyltransferase | Humans | inhibitor |
ADME / PK
| Absorption | Eliglustat administered in multiple doses of 84 mg twice daily had a C<sub>max</sub> of 12.1 to 25.0 ng/mL in CYP2D6 extensive metabolizers (EMs), 44.6 ng/mL in CYP2D6 intermediate metabolizers (IMs), and 113 to 137 ng/mL in CYP2D6 poor metabolizers (PMs). The median T<sub>max</sub> was 1.5-2 hr in CYP2D6 EMs, 2 hr in CYP2D6 IMs, and 3 hr in CYP2D6 PMs. The AUC<sub>tau</sub> was 76.3-143 ng∙hr/mL in CYP2D6 EMs, 306 ng∙hr/mL in CYP2D6 IMs, and 922-1057 ng∙hr/mL in CYP2D6 PMs. In CYP2D6 EMs, the pharmacokinetics of eliglustat is time-dependent, and for doses that range between 42 and 294 mg, exposure increases in a more than dose-proportional fashion. In CYP2D6 PMs, eliglustat pharmacokinetics is linear and time-independent. In a steady state, the systemic exposure of 84 mg eliglustat twice daily is 7- to 9-fold higher in CYP2D6 PMs compared to EMs. Following the oral administration of a single 84 mg dose of eliglustat, bioavailability in CYP2D6 EMs was lower than 5%. The low oral bioavailability of eliglustat suggests the role of transporters and/or an extensive first-pass metabolism. Eliglustat can be taken with or without food. In CYP2D6 EMs, severe renal impairment did not have an effect on eliglustat pharmacokinetics. The effect of renal impairment on eliglustat pharmacokinetics was not evaluated in CYP2D6 IMs, CYP2D6 PMs or CYP2D6 EMs with end-stage renal failure. Compared to CYP2D6 EMs with normal hepatic function, C<sub>max</sub> and AUC were 1.2-fold higher in CYP2D6 EMs with mild hepatic impairment, while C<sub>max</sub> and AUC were 2.8- and 5.2-fold higher, respectively, in CYP2D6 EMs with moderate hepatic impairment. The effect of mild and moderate hepatic impairment in CYP2D6 IMs and PMs, and the effect of severe hepatic impairment were not evaluated. |
|---|---|
| Half-life | Eliglustat has a terminal elimination half-life of 6.5 hours in CYP2D6 extensive metabolizers (EMs) and 8.9 h in CYP2D6 poor metabolizers (PMs). |
| Protein binding | In plasma, the protein binding of eliglustat goes from 76% to 83%. |
| Metabolism | Eliglustat is mostly metabolized by CYP2D6, and to a lower extent, by CYP3A4. In patients that are CYP2D6 poor metabolizers (PMs), eliglustat is mainly metabolized by CYP3A4. The primary metabolic pathways of eliglustat involve the sequential oxidation of the octanoyl moiety and the 2,3-dihydro-1,4-benzodioxane moiety. The combination of these two pathways results in the production of several oxidative metabolites. After evaluating the potency of eliglustat metabolites, it was determined that none of them were active. Genz-399240 (M24) was identified as the major metabolite of eliglustat, while the rest of the metabolites contributed to less than 10% of total drug-related exposures. Genz-399240 (M24) did not show any major off-target effects; therefore, a transporter substrate specificity characterization was not performed. |
| Route of elimination | Eliglustat is mainly excreted in urine (42%) and feces (51%) as metabolites after oral administration. |
| Volume of distribution | In CYP2D6 extensive metabolizers (EM), the volume of distribution of eliglustat administered IV was 835 L. |
| Clearance | In healthy CYP2D6 extensive metabolizers (EMs) administered 42 mg of eliglustat IV (0.5 times the recommended oral dose), clearance was 88 L/h (80-105 L/h). |
Formulation & handling
- Eliglustat is a small molecule oral capsule formulation with moderate lipophilicity and low water solubility.
- Avoid co-administration with grapefruit products and St. John's Wort due to CYP3A-mediated interactions affecting drug metabolism.
- Stable for oral dosing with or without food; high-fat meals may slightly reduce peak plasma concentrations without clinical impact.
Regulatory status
| Lifecycle | The API is marketed in the US, Canada, and the EU, with key US patents having expired in April 2022, while a later US patent remains active until January 2031, indicating a partially mature market with ongoing patent protection in the United States. |
|---|
| Markets | US, Canada, EU |
|---|
Supply Chain
| Supply chain summary | Eliglustat is primarily originated by a single company with branded products marketed across the US, Canada, and the EU. Multiple US patents protect the product, with the latest expiration date in January 2031, indicating that significant generic competition has not yet commenced but may emerge following patent expiry. The current patent landscape supports continued branded exclusivity in key markets. |
|---|
Safety
| Toxicity | Eliglustat overdose may manifest as dizziness marked by disequilibrium, hypotension, bradycardia, nausea, and vomiting. These symptoms were detected in a healthy subject taking 21-times the dose recommended to type 1 Gaucher disease patients. Eliglustat has no known antidote. In case of acute overdose, the patient should be carefully observed and given symptomatic and supportive treatment. Due to the large volume of distribution of eliglustat, hemodialysis is not likely to be beneficial. Acute dose toxicity studies were performed in rats and dogs. In rats, the maximum tolerated dose was 200 mg/kg, and in non-fasted dogs, the maximum tolerated dose was 25 mg/kg. Some of the adverse effects detected in these toxicity studies manifested on the GI tract, hematology parameters related to hemoglobin and coagulation process, reproductive organs, thymus and other lymphoid organs. Adverse effects in the kidney and liver were only detected in rats. Carcinogenic studies were performed in both Sprague-Dawley rats and CD-1 mice. In doses up to 50 mg/kg/day in female Sprague-Dawley rats and 75 mg/kg/day in male Sprague-Dawley rats and CD-1 mice, eliglustat did not induce neoplasms. Eliglustat was negative in the following mutagenesis tests: Ames test, chromosome aberration test in human peripheral blood lymphocytes, mouse lymphoma gene mutation assay and in vivo oral mouse micronucleus test. |
|---|
High Level Warnings:
- Eliglustat overdose can cause dizziness, hypotension, bradycardia, nausea, and vomiting
- No specific antidote exists, and treatment is supportive
- Due to Eliglustat's large volume of distribution, hemodialysis is ineffective for overdose management
Eliglustat is a type of Other enzyme replacements/modifiers
Others
Eliglustat (Other enzyme replacements/modifiers), classified under Enzyme Replacements/modifiers
Enzyme replacements/modifiers are a crucial category of pharmaceutical active pharmaceutical ingredients (APIs) utilized in the treatment of various enzyme-related disorders. Enzymes play a vital role in the normal functioning of the body by catalyzing specific biochemical reactions. However, in certain medical conditions, the body may lack or produce dysfunctional enzymes, leading to serious health complications.
Enzyme replacement therapy (ERT) involves administering exogenous enzymes to compensate for the enzyme deficiency in patients. These enzymes are typically derived from natural sources or produced using recombinant DNA technology. By introducing these enzymes into the body, they can effectively substitute the missing or defective enzymes, thereby restoring normal metabolic processes.
On the other hand, enzyme modifiers are API substances that regulate the activity of specific enzymes within the body. These modifiers can either enhance or inhibit the enzyme's function, depending on the therapeutic objective. By modulating enzyme activity, these APIs can restore the balance of enzymatic reactions, leading to improved physiological outcomes.
Enzyme replacements/modifiers have shown remarkable success in treating various genetic disorders, such as Gaucher disease, Fabry disease, and lysosomal storage disorders. Additionally, they have demonstrated potential in managing enzyme deficiencies associated with rare diseases and certain types of cancer.
The development and production of enzyme replacements/modifiers involve rigorous research, formulation optimization, and adherence to stringent quality control measures. Pharmaceutical companies invest substantial resources in developing these APIs to ensure their safety, efficacy, and compliance with regulatory standards.
Overall, enzyme replacements/modifiers represent a vital therapeutic category in modern medicine, offering hope and improved quality of life for patients with enzyme-related disorders.
Eliglustat API manufacturers & distributors
Compare qualified Eliglustat API suppliers worldwide. We currently have 5 companies offering Eliglustat 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 |
|---|---|---|---|---|---|
| Biophore India | Producer | India | India | CoA, USDMF | 46 products |
| Cipla | Producer | India | India | CoA, USDMF | 164 products |
| Dr. Reddy's | Producer | India | India | BSE/TSE, CoA, FDA, GMP, MSDS, USDMF, WC | 170 products |
| Maithri Labs. | Producer | India | India | CoA, USDMF | 5 products |
| Raks Pharma | Producer | India | India | CoA, USDMF | 58 products |
When sending a request, specify which Eliglustat API quality you need: for example EP (Ph. Eur.), USP, JP, BP, or another pharmacopoeial standard, as well as the required grade (base, salt, micronised, specific purity, etc.).
Use the list above to find high-quality Eliglustat API suppliers. For example, you can select GMP, FDA or ISO certified suppliers. Visit our help page to learn more about sourcing APIs via Pharmaoffer.
