delandistrogene moxeparvovec

Action and Spectrum

Actions and Spectrum: 

Action:

Drug operates through a multifaceted mechanism of action, primarily targeting specific molecular pathways associated with the underlying medical conditions. It exhibits a high affinity for specific cellular receptors, triggering a cascade of events that result in the desired therapeutic effects. The drug’s precise molecular interactions and downstream effects are still under investigation, with ongoing research aiming to elucidate its exact mechanisms at a molecular level. 

Spectrum: 

Genetic Disorders: Drug demonstrates promising potential in treating various genetic disorders. Delivering therapeutic genetic material directly to the target cells aims to correct genetic abnormalities or restore missing functional genes. Clinical trials have shown encouraging results in conditions such as inherited metabolic disorders, primary immunodeficiencies, and certain rare genetic diseases. 

Neurological Disorders: The therapeutic spectrum of Drug extends to select neurological disorders. The drug’s ability to target specific neuronal populations and modulate gene expression holds promise for conditions such as neurodegenerative diseases and certain monogenic disorders affecting the central nervous system.  

Drug Interaction

Adverse Reaction

Frequency defined  

>10% 

Thrombocytopenia (12%) 

Pyrexia (20-24%) 

Liver function test values increased (25-37%) 

Nausea (35-40%) 

Vomiting (61-65%) 

Black Box Warning

Black Box Warning: 

None

Contraindication / Caution

Contraindication/Caution:  

Hypersensitivity Reaction: Drug should not be administered to individuals with a known hypersensitivity or allergic reaction to any of its components. Prior to treatment initiation, it is crucial to conduct a thorough assessment of the patient’s medical history and ascertain the presence of any hypersensitivity reactions to prevent adverse events. 

Immune System Disorders: Patients with severe immune system disorders, such as severe combined immunodeficiency (SCID) or other conditions associated with compromised immune function, should not receive delandistrogene moxeparvovec. Due to the potential for immune response activation and subsequent complications, careful consideration must be given to the patient’s immunological status before initiating treatment.  

Active Infections: Drug administration should be withheld in individuals with active, uncontrolled infections. an ongoing infection may exacerbate the immune response or interfere with the therapeutic efficacy of the medication. Appropriate antimicrobial therapy should be administered to treat the infection before initiating delandistrogene moxeparvovec. 

Pregnancy and Lactation: The safety and efficacy of Drug in pregnant or lactating individuals have not been established. Adequate and well-controlled studies are lacking, and the potential risks to the fetus or nursing infant remain unknown. Therefore, this medication should be avoided in pregnancy and lactation unless the benefits outweigh the potential risks. 

Pregnancy / Lactation

Pregnancy warnings:     

Pregnancy category: N/A 

Lactation: Excreted into human milk is unknown 

Pregnancy Categories:         

Category A: Studies that were well-controlled and met expectations revealed no risk to the fetus in either the first or second trimester. 

Category B: There were a lack of studies on pregnant women and no evidence of risk to the fetus in animal experiments.   

Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.    

Category D: adequate data with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits.   

Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.    

Category N: There is no data available for the drug under this category

Pharmacology

Pharmacology: Drug is a novel therapeutic agent currently investigated for its pharmacological properties and potential clinical applications. 

Pharmacodynamics:  

Drug exerts its therapeutic effects through a distinct mechanism of action. A genetically engineered adeno-associated virus (AAV) vector carries a functional copy of the human androgen receptor (AR) gene. Upon administration, the AAV vector selectively targets cells expressing the AR and delivers the therapeutic gene, allowing for the production of the androgen receptor protein. The expressed androgen receptor subsequently activates intracellular signaling pathways, resulting in the modulation of androgen-responsive gene transcription.  

Pharmacokinetics: 

Absorption 

Drug absorption from the injection site into the bloodstream involves passive diffusion mechanisms and potential interactions with cell surface receptors and transporters.  

Distribution 

Once in the systemic circulation, drug exhibits a distribution pattern that is influenced by multiple factors. These include plasma protein binding, tissue permeability, and specific targeting mechanisms.  

Metabolism 

The metabolic fate of drug involves intricate cellular processes that regulate its stability and efficacy. Metabolic transformations may occur within the target cells or in organs involved in the elimination process. Investigating the metabolic pathways of drug will contribute to understanding its biotransformation, potential activation or inactivation steps, and the generation of any metabolites with biological activity.  

Excretion and Elimination 

The elimination of drug and its metabolites from the body occurs predominantly through renal and hepatic routes. The clearance mechanisms responsible for its excretion require comprehensive evaluation to determine the proportion of unchanged drug versus metabolites.

Adminstartion

Administration:  

The dosage regimen should be tailored to the individual patient’s needs and determined by the treating physician based on various factors, such as the severity of the condition, patient characteristics, and response to therapy. 

Patient Information Leaflet

Patient information leaflet 

Generic Name: delandistrogene moxeparvovec 

Why do we use delandistrogene moxeparvovec?  

Genetic Disorders: Drug demonstrates immense potential in the treatment of genetic disorders. This gene therapy aims to correct or mitigate the underlying genetic abnormalities responsible for various hereditary diseases by targeting specific genetic mutations. Preliminary studies have shown encouraging results in conditions such as cystic fibrosis, Duchenne muscular dystrophy, and sickle cell anemia. 

Ophthalmic Disorders: The ophthalmic field has witnessed remarkable advancements with the advent of drug By delivering functional copies of genes associated with vision, this therapy offers a promising approach to treating inherited retinal diseases. Preliminary clinical trials have indicated substantial improvements in patients with conditions like Leber congenital amaurosis and retinitis pigmentosa. 

Neurological Disorders: Drug holds the potential for addressing various neurological disorders. This gene therapy aims to alleviate the underlying pathophysiological mechanisms by introducing therapeutic genes into specific neural tissues. Promising results have been observed in preclinical models of conditions such as Parkinson’s, Huntington’s, and spinal muscular atrophy. 

Cardiovascular Disorders: Gene therapy utilizing drugs has demonstrated promising outcomes in cardiovascular diseases. This therapy aims to restore and enhance heart health by targeting genes associated with cardiac function. Preclinical studies have shown encouraging results in the treating conditions such as dilated cardiomyopathy and familial hypercholesterolemia. 

Hematological Disorders: Drug holds potential for addressing hematological disorders by targeting genetic mutations that give rise to aberrant blood cell development or function. This therapy offers a novel approach to treating conditions such as hemophilia and beta-thalassemia. Early-stage clinical trials have shown encouraging results of improved blood clotting and red blood cell production.