modelElivaldogeneAutotemcel
Extends from Pharmacolibrary.Drugs.ATC.A.A16AX21.
Information
| name: | ElivaldogeneAutotemcel | |
| ATC code: | A16AX21 | route: | intravenous |
| compartments: | 0 | |
| dosage: | 5.9 | mg |
| volume of distribution: | 1 | L |
| clearance: | 0 | |
| other parameters in model implementation | ||
Elivaldogene autotemcel (also known as Lenti-D) is an autologous hematopoietic stem cell gene therapy used for the treatment of cerebral adrenoleukodystrophy (CALD). It is intended to add a functional copy of the ABCD1 gene to the patient's own hematopoietic stem cells, thereby enabling the production of the ALD protein which is deficient in this condition. The therapy is indicated for boys with early active cerebral adrenoleukodystrophy and has received conditional approval in the EU, but was withdrawn from the market for commercial reasons. It is not currently approved in the US.
Pharmacokinetics
No direct pharmacokinetic (PK) parameters (absorption, distribution, metabolism, elimination) are reported for elivaldogene autotemcel, as it is a genetically modified autologous cell therapy. Its fate is determined by engraftment and persistence of the modified stem cells, not by traditional PK processes. No studies provide standard PK data. Data is applicable to pediatric males with CALD receiving a single intravenous dose following myeloablative conditioning.
References
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 |
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)