Lumasiran: Difference between revisions

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== '''What Is Lumasiran?''' ==
{{Short description|An RNA interference therapeutic for primary hyperoxaluria type 1}}


*Lumasiran (OXLUMO) is a HAO1-directed small interfering ribonucleic acid ([[siRNA]]) indicated for the treatment of [[primary hyperoxaluria type 1]] (PH1) to lower urinary [[oxalate]] levels in pediatric and adult patients.
==Lumasiran==
[[File:Lumasiran_02.svg|thumb|right|Chemical structure of Lumasiran]]
'''Lumasiran''' is a medication used in the treatment of [[primary hyperoxaluria type 1]] (PH1), a rare genetic disorder characterized by the overproduction of [[oxalate]], leading to kidney stones and renal failure. Lumasiran is an RNA interference (RNAi) therapeutic that targets the [[hydroxyacid oxidase 1]] (HAO1) gene, which encodes the enzyme glycolate oxidase. By reducing the production of glycolate oxidase, Lumasiran decreases the conversion of glycolate to glyoxylate, thereby reducing the production of oxalate.


<youtube>
==Mechanism of Action==
title='''{{PAGENAME}}'''
Lumasiran works by utilizing the natural cellular process of RNA interference to silence the expression of the HAO1 gene. This gene is responsible for the production of glycolate oxidase, an enzyme that plays a crucial role in the metabolic pathway leading to oxalate production. By inhibiting this enzyme, Lumasiran effectively reduces the levels of oxalate in the body, thereby mitigating the symptoms and complications associated with primary hyperoxaluria type 1.
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== '''What are the uses of this medicine?''' ==
==Administration and Dosage==
Lumasiran is administered via subcutaneous injection. The dosing regimen typically involves an initial loading phase followed by maintenance doses. The specific dosage and frequency depend on the patient's body weight and the severity of the condition. It is important for healthcare providers to monitor patients regularly to adjust the dosage as needed and to assess the therapeutic response.


* This medicine is is indicated for the treatment of [[primary hyperoxaluria type 1]] (PH1) to lower urinary [[oxalate]] levels in pediatric and adult patients.
==Side Effects==
Common side effects of Lumasiran include injection site reactions, such as redness, swelling, and pain. Some patients may also experience gastrointestinal symptoms, such as nausea and abdominal pain. It is important for patients to report any adverse effects to their healthcare provider, as these may require medical attention or adjustment of the treatment regimen.


== '''How does this medicine work?''' ==
==Clinical Trials==
* Lumasiran '''reduces levels of glycolate oxidase (GO) enzyme''' by targeting the hydroxyacid oxidase 1 (HAO1) messenger ribonucleic acid (mRNA) in hepatocytes through RNA interference.
Lumasiran has undergone extensive clinical trials to evaluate its safety and efficacy in patients with primary hyperoxaluria type 1. These trials have demonstrated significant reductions in urinary oxalate levels, which correlate with a decrease in the risk of kidney stone formation and renal damage. The results of these trials have supported the approval of Lumasiran for the treatment of PH1.
* '''Decreased GO enzyme levels reduce the amount of available glyoxylate, a substrate for oxalate production'''.  
* OXLUMO is not expected to be effective in primary hyperoxaluria type 2 (PH2) or type 3 (PH3) because its mechanism of action does not affect the metabolic pathways causing hyperoxaluria in PH2 and PH3.


== '''Who Should Not Use this medicine ?''' ==
==Related pages==
* [[Primary hyperoxaluria]]
* [[RNA interference]]
* [[Kidney stone]]
* [[Genetic disorder]]


* OXLUMO is not expected to be effective in [[primary hyperoxaluria type 2]] (PH2) or [[Primary hyperoxaluria type iii|type 3 (PH3)]]
[[Category:RNA interference therapies]]
 
[[Category:Genetic disorders]]
== '''Is this medicine FDA approved?''' ==
[[Category:Kidney diseases]]
 
* It was approved for use in the United States in 2020.
 
== '''How should this medicine be used?''' ==
 
* The recommended dose of OXLUMO by subcutaneous injection is based on body weight.
 
'''For body weight less than 10 kg:'''
* Loading Dose is 6 mg/kg once monthly for 3 doses
* Maintenance Dose(begin 1 month after the last loading dose) is 3 mg/kg once monthly
 
'''For body weight 10 kg to less than 20 kg:'''
* Loading Dose is 6 mg/kg once monthly for 3 doses
* Maintenance Dose(begin 1 month after the last loading dose) is 6 mg/kg once every 3 months (quarterly)
 
'''For body weight 20 kg and above:'''
* Loading Dose is  3 mg/kg once monthly for 3 doses
* Maintenance Dose(begin 1 month after the last loading dose) is 3 mg/kg once every 3 months (quarterly)
 
'''Administration'''
* OXLUMO is intended for subcutaneous use and should be administered by a healthcare professional.
* Administer subcutaneous injection into the abdomen, thigh, or the side or back of the upper arms.
* Rotate injection sites.
* Do not inject into scar tissue or areas that are reddened, inflamed, or swollen.
* If injecting into the abdomen, avoid the area around the navel.
* If more than one injection is needed for a single dose of OXLUMO, the injection sites should be at least 2 cm apart.
* Discard unused portion of the drug.
 
== '''What are the  dosage forms and brand names of this medicine?''' ==
 
This medicine is available in fallowing doasage form:
'''As an Injection:''' 94.5 mg/0.5 mL in a single-dose vial.
 
This medicine is available in fallowing brand namesː
* '''OXLUMO'''
 
=='''What side effects can this medication cause?'''==
 
Common possible side effects of this medicine include:
* [[Injection site reaction]]
* Abdominal pain
 
==''' What special precautions should I follow?''' ==
 
 
== '''What to do in case of emergency/overdose?''' ==
 
== '''Can this medicine be used in pregnancy?''' ==
 
* There are no available data with the use of OXLUMO in pregnant women to evaluate a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.
 
=='''Can this medicine be used in children?'''==
 
* The safety and effectiveness of OXLUMO have been established in pediatric patients aged birth and older.
 
== '''What should I know about storage and disposal of this medication?''' ==
 
* Store at 2°C to 25°C [36°F to 77°F].
* Store OXLUMO in its original container until ready for use.
 
{{Other alimentary tract and metabolism products}}
{{Portal bar | Medicine}}
 
[[Category:Breakthrough therapy]]
[[Category:Orphan drugs]]
{{coststubd}}

Latest revision as of 03:56, 13 February 2025

An RNA interference therapeutic for primary hyperoxaluria type 1


Lumasiran[edit]

Chemical structure of Lumasiran

Lumasiran is a medication used in the treatment of primary hyperoxaluria type 1 (PH1), a rare genetic disorder characterized by the overproduction of oxalate, leading to kidney stones and renal failure. Lumasiran is an RNA interference (RNAi) therapeutic that targets the hydroxyacid oxidase 1 (HAO1) gene, which encodes the enzyme glycolate oxidase. By reducing the production of glycolate oxidase, Lumasiran decreases the conversion of glycolate to glyoxylate, thereby reducing the production of oxalate.

Mechanism of Action[edit]

Lumasiran works by utilizing the natural cellular process of RNA interference to silence the expression of the HAO1 gene. This gene is responsible for the production of glycolate oxidase, an enzyme that plays a crucial role in the metabolic pathway leading to oxalate production. By inhibiting this enzyme, Lumasiran effectively reduces the levels of oxalate in the body, thereby mitigating the symptoms and complications associated with primary hyperoxaluria type 1.

Administration and Dosage[edit]

Lumasiran is administered via subcutaneous injection. The dosing regimen typically involves an initial loading phase followed by maintenance doses. The specific dosage and frequency depend on the patient's body weight and the severity of the condition. It is important for healthcare providers to monitor patients regularly to adjust the dosage as needed and to assess the therapeutic response.

Side Effects[edit]

Common side effects of Lumasiran include injection site reactions, such as redness, swelling, and pain. Some patients may also experience gastrointestinal symptoms, such as nausea and abdominal pain. It is important for patients to report any adverse effects to their healthcare provider, as these may require medical attention or adjustment of the treatment regimen.

Clinical Trials[edit]

Lumasiran has undergone extensive clinical trials to evaluate its safety and efficacy in patients with primary hyperoxaluria type 1. These trials have demonstrated significant reductions in urinary oxalate levels, which correlate with a decrease in the risk of kidney stone formation and renal damage. The results of these trials have supported the approval of Lumasiran for the treatment of PH1.

Related pages[edit]

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