| Size | Price | |
|---|---|---|
| 500mg | ||
| 1g | ||
| Other Sizes |
| ADME/Pharmacokinetics |
Absorption, Distribution and Excretion
In patients with non-end-stage renal disease, the Cmax of agarsidase α was 3710 ± 855 U/mL, and the AUC was 256,958 ± 63,499 minU/mL. Following non-specific proteolysis, amino acids in the protein drug can be reused for protein synthesis or further broken down and excreted via the kidneys. In patients with non-end-stage renal disease, the steady-state volume of distribution is approximately 17% of body weight, regardless of sex. At doses of 0.007–0.2 mg/kg, the clearance rate was 2.66 mL/min/kg in males and 2.10 mL/min/kg in females. Metabolism/Metabolites Data on the metabolism of agarsidase α are currently unavailable. However, protein drugs are expected to be degraded into smaller peptides and amino acids by proteases and other catalytic enzymes. Biological half-life The elimination half-life for males is 108 ± 17 minutes, and for females it is 89 ± 28 minutes. |
|---|---|
| Toxicity/Toxicokinetics |
Effects During Pregnancy and Lactation
◉ Overview of Use During Lactation Since there is no published experience regarding the use of eligliflozin during lactation, alternative medications may be preferred, especially when breastfeeding newborns or premature infants. ◉ Effects on Breastfed Infants No published information found as of the revision date. ◉ Effects on Lactation and Breast Milk No published information found as of the revision date. Protein Binding Agarsidase α is not expected to bind to proteins in circulation. |
| References |
[1]. Keating GM. Agalsidase alfa: a review of its use in the management of Fabry disease. BioDrugs. 2012 Oct 1;26(5):335-54.
|
| Additional Infomation |
Eliglustat tartrate is a tartrate salt, specifically the hemitartrate salt of ligorupstat. It is a ceramide glucosyltransferase inhibitor (in its tartrate form) used to treat Gaucher disease. It is an EC 2.4.1.80 (ceramide glucosyltransferase) inhibitor. It contains ligorupstat (1+). Agardilase Alpha is a recombinant human α-galactosidase A, similar to agarsidase Beta. While patients generally do not experience a significant difference in efficacy between the two drugs, some patients may benefit more from agarsidase Beta. Following a contamination incident in 2009, the use of agarsidase Beta declined in Europe, and agarsidase Alpha was adopted instead. Agardilase Alpha was approved by the EMA on August 3, 2001. See also: Eliglustat (with the active moiety); Agardilase Beta (note moved to).
Drug Indications Agarsidase Alpha is indicated for the treatment of Fabry disease. Repula is indicated for long-term enzyme replacement therapy in patients diagnosed with Fabry disease (α-galactosidase A deficiency). Mechanism of Action α-galactosidase A is taken up by cells via mannose-6-phosphate receptors. Agardase α hydrolyzes globular trihexosylceramide and other glycosphingolipids, which are normally hydrolyzed by endogenous α-galactosidase A. Preventing the accumulation of glycosphingolipids can prevent or alleviate symptoms of Fabry disease, such as renal failure, cardiomyopathy, or cerebrovascular events. Pharmacodynamics Agardase α is a recombinant human α-galactosidase A used as enzyme replacement therapy for Fabry disease. It is characterized by a long duration of action and a wide therapeutic index. Patients should be informed of the risks of infusion-related reactions and hypersensitivity reactions. |
| Molecular Weight |
959.188g/mol
|
|---|---|
| Exact Mass |
958.551
|
| CAS # |
104138-64-9
|
| PubChem CID |
52918379
|
| Appearance |
Typically exists as solid at room temperature
|
| Hydrogen Bond Donor Count |
8
|
| Hydrogen Bond Acceptor Count |
16
|
| Rotatable Bond Count |
25
|
| Heavy Atom Count |
68
|
| Complexity |
617
|
| Defined Atom Stereocenter Count |
6
|
| SMILES |
CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@@H](C2=CC3=C(C=C2)OCCO3)O.CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@@H](C2=CC3=C(C=C2)OCCO3)O.[C@@H]([C@H](C(=O)O)O)(C(=O)O)O
|
| InChi Key |
KUBARPMUNHKBIQ-VTHUDJRQSA-N
|
| InChi Code |
InChI=1S/2C23H36N2O4.C4H6O6/c2*1-2-3-4-5-6-9-22(26)24-19(17-25-12-7-8-13-25)23(27)18-10-11-20-21(16-18)29-15-14-28-20;5-1(3(7)8)2(6)4(9)10/h2*10-11,16,19,23,27H,2-9,12-15,17H2,1H3,(H,24,26);1-2,5-6H,(H,7,8)(H,9,10)/t2*19-,23-;1-,2-/m111/s1
|
| Chemical Name |
N-[(1R,2R)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-pyrrolidin-1-ylpropan-2-yl]octanamide;(2R,3R)-2,3-dihydroxybutanedioic acid
|
| HS Tariff Code |
2934.99.9001
|
| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
|
| Solubility (In Vitro) |
May dissolve in DMSO (in most cases), if not, try other solvents such as H2O, Ethanol, or DMF with a minute amount of products to avoid loss of samples
|
|---|---|
| Solubility (In Vivo) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80： Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 ：Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400 (Please use freshly prepared in vivo formulations for optimal results.) |
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease
CTID: NCT05698901
Phase: Status: Recruiting
Date: 2023-11-18
Products are for research use only; We do not sell to patients
Copyright 2020 InvivoChem LLC | All Rights Reserved
