modelMigalastat
Extends from Pharmacolibrary.Drugs.ATC.A.A16AX14.
Information
| name: | Migalastat | |
| ATC code: | A16AX14 | route: | oral |
| compartments: | 1 | |
| dosage: | 150 | mg |
| volume of distribution: | 89 | L |
| clearance: | 12.5 | L/h |
| other parameters in model implementation | ||
Migalastat is an oral pharmacological chaperone used for the treatment of Fabry disease—a rare X-linked lysosomal storage disorder caused by deficiency of the enzyme alpha-galactosidase A (GLA). Migalastat stabilizes specific mutant forms of GLA, increasing their trafficking to lysosomes and thus enhancing enzymatic activity. Migalastat is approved for use in several regions including the EU, USA, and Japan.
Pharmacokinetics
Pharmacokinetic parameters following oral administration of migalastat 150 mg to healthy adult volunteers (mean age approx. 30 years, both sexes) under fasting conditions.
References
Leonowens, C, et al., & Johnson, FK (2022). Population Pharmacokinetics of Oral Migalastat in Adolescents and Adults With and Without Renal Impairment. Clinical pharmacology in drug development 11(12) 1367–1381. DOI:10.1002/cpdd.1160 PUBMED:https://pubmed.ncbi.nlm.nih.gov/36331497
McCafferty, EH, & Scott, LJ (2019). Migalastat: A Review in Fabry Disease. Drugs 79(5) 543–554. DOI:10.1007/s40265-019-01090-4 PUBMED:https://pubmed.ncbi.nlm.nih.gov/30875019
Ino, H, et al., & Hirama, T (2013). Pharmacokinetics, safety, and tolerability following single-dose migalastat hydrochloride (GR181413A/AT1001) in healthy male Japanese subjects. Journal of drug assessment 2(1) 87–93. DOI:10.3109/21556660.2013.827117 PUBMED:https://pubmed.ncbi.nlm.nih.gov/27536442
Parameters
| Type | Name | Default | Description |
|---|---|---|---|
| Modelica.Units.SI.Mass | weight (from PK_1C) | 75 | patient weight (kg) |
| Modelica.Units.SI.SpecificVolume | VdPerKg (from PK_1C) | 0.9 | Volume of distribution (L/kg) |
| Modelica.Units.SI.MassFraction | F (from PK_1C) | 0.8 | bioavailiability (0-1) |
| Pharmacolibrary.Types.Clearance | Cl (from PK_1C) | 20 | clearance |
| Modelica.Units.SI.Time | adminTime (from PK_1C) | 60 | first administration time (s) |
| Modelica.Units.SI.Time | adminDuration (from PK_1C) | 600 | administration duration (s) |
| Modelica.Units.SI.Time | adminPeriod (from PK_1C) | 8*60*60 | period of administration (default 8 hours)(s) |
| Pharmacolibrary.Types.Mass | adminMass (from PK_1C) | 1000 | administration mass (mg) |
| Integer | adminCount (from PK_1C) | 8 | number of dose administered (1) |
| Pharmacolibrary.Types.Volume | Vd (from PK_1C) | VdPerKg*weight | Volume of distribution (m3) |
| Pharmacolibrary.Types.MassConcentration | Cmin (from PK_1C) | 0.004 | minimal therapeutic range |
| Pharmacolibrary.Types.MassConcentration | Cmax (from PK_1C) | 0.008 | minimal therapeutic range |
| Pharmacolibrary.Types.MassConcentration | Ctox_peak (from PK_1C) | 0.012 | toxicity peak level |
| Pharmacolibrary.Types.MassConcentration | Ctox_trough (from PK_1C) | 0.006 | toxicity trough level |
| Pharmacolibrary.Types.TransferRate | ka (from PK_1C_enteral) | 0.016666666666666666 | first order absorption rate |
| Modelica.Units.SI.Time | Tlag (from PK_1C_enteral) | 600 | delay between oral administration and absorption (default 10min) |
Connectors
| Type | Name | Default | Description |
|---|---|---|---|
| Types.ConcentrationOutput | C_central (from PK_1C) | ||
| Interfaces.ConcentrationPort_b | centralCPort (from PK_1C) |
Components
| Type | Name | Default | Description |
|---|---|---|---|
| Pharmacokinetic.NoPerfusedTissueCompartment | central (from PK_1C) | ||
| Pharmacokinetic.ClearanceDrivenElimination | elim (from PK_1C) | ||
| Sources.PeriodicDose | periodicDose (from PK_1C) | ||
| Modelica.Units.SI.Time | t1_2 (from PK_1C) | elimination half-life |
Revisions
- 06/2025 Tomas Kulhanek, generated model from data extracted from PUBMED, DrugBank and LLM(GPT4.1)