Viltolarsen: Difference between revisions

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=='''What Is Viltolarsen?'''==
{{Short description|An article about the drug Viltolarsen}}
Viltolarsen (VILTEPSO) is an [[antisense]] [[oligonucleotide]] indicated for the treatment of [[Duchenne muscular dystrophy]] (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
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'''Viltolarsen''' is a [[pharmaceutical drug]] used in the treatment of [[Duchenne muscular dystrophy]] (DMD), a severe type of [[muscular dystrophy]] characterized by rapid progression of muscle degeneration. Viltolarsen is specifically designed to target mutations in the [[dystrophin]] gene that are amenable to exon 53 skipping.


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==Mechanism of Action==
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Viltolarsen is an [[antisense oligonucleotide]] that works by binding to exon 53 of the [[dystrophin]] pre-mRNA. This binding causes the exon to be skipped during [[mRNA splicing]], resulting in the production of a truncated but functional dystrophin protein. This mechanism is intended to partially restore the function of the dystrophin protein in patients with specific genetic mutations.
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==Clinical Use==
=='''What are the uses of this medicine?'''==
Viltolarsen is indicated for the treatment of patients with Duchenne muscular dystrophy who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. It is administered via [[intravenous infusion]] and is typically given on a weekly basis.


*This medicine is indicated for the treatment of [[Duchenne muscular dystrophy]] (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
==Side Effects==
*This indication is approved under [[accelerated approval]] based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO .
Common side effects of Viltolarsen include [[injection site reactions]], [[upper respiratory tract infections]], and [[fever]]. Patients receiving Viltolarsen should be monitored for potential adverse reactions, and the drug should be used with caution in patients with [[renal impairment]].
*Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
 
=='''How does this medicine work?'''==


*VILTEPSO is designed to '''bind to exon 53 of [[dystrophin]] pre-mRNA resulting in exclusion of this exon during [[mRNA]] processing in patients with genetic mutations that are amenable to exon 53 skipping'''.
==Development and Approval==
*Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.
Viltolarsen was developed by [[Nippon Shinyaku]] and [[NS Pharma]]. It received accelerated approval from the [[United States Food and Drug Administration]] (FDA) in 2020 based on its ability to increase dystrophin production in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.


=='''Who Should Not Use this medicine ?'''==
==Related pages==
* [[Duchenne muscular dystrophy]]
* [[Antisense oligonucleotide]]
* [[Dystrophin]]
* [[Exon skipping]]


*This medicine have no usage limitations.
[[Category:Antisense oligonucleotides]]
 
[[Category:Drugs for muscular dystrophy]]
=='''Is this medicine FDA approved?'''==
 
*It was approved for use in the United States in 2020.
 
=='''How should this medicine be used?'''==
 
*Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VILTEPSO.
*Consider measurement of [[glomerular filtration rate]] prior to initiation of VILTEPSO.
*Monitoring for kidney toxicity during treatment is recommended.
*Obtain the urine samples prior to infusion of VILTEPSO or at least 48 hours after the most recent infusion.
*The '''recommended dosage of VILTEPSO is 80 mg/kg administered once weekly as a 60-minute intravenous infusion'''.
*If a dose of VILTEPSO is missed, it should be administered as soon as possible after the scheduled dose time.
 
 
'''Administration'''
 
*VILTEPSO is administered via intravenous infusion using a peripheral or central venous catheter.
*Flush the intravenous access line with 0.9% Sodium Chloride Injection, USP, after infusion.
*Filtration of VILTEPSO is not required.
*Infuse VILTEPSO over 60 minutes.
*Do not mix other medications with VILTEPSO or infuse other medications concomitantly via the same intravenous access line.
*VILTEPSO should be mixed with 0.9% Sodium Chloride Injection, USP, only.
 
=='''What are the  dosage forms and brand names of this medicine?'''==
 
This medicine is available in fallowing doasage form:
 
*'''As Injection:''' 250 mg/5 mL (50 mg/mL) solution in a single-dose vial
 
This medicine is available in fallowing brand namesː
 
*'''VILTEPSO'''
 
=='''What side effects can this medication cause?'''==
 
Common possible side effects of this medicine include:
 
*Upper respiratory tract infection
*[[Injection site reaction]]
*Cough
*[[Pyrexia]]
*[[Contusion]]
*[[Arthralgia]]
*Diarrhea
*Vomiting
*Abdominal pain
*Ejection fraction decreased
*[[Urticaria]]
 
==''' What special precautions should I follow?'''==
 
*This medicine may cause kidney toxicity.
*Kidney function should be monitored; [[creatinine]] may not be a reliable measure of renal function in DMD patients.
 
=='''What to do in case of emergency/overdose?'''==
{{overdose}}
 
=='''Can this medicine be used in pregnancy?'''==
 
*There are no human or animal data available to assess the use of VILTEPSO during pregnancy.
 
=='''Can this medicine be used in children?'''==
 
*VILTEPSO is indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping, '''including pediatric patients'''.
 
=='''What should I know about storage and disposal of this medication?'''==
 
*Store VILTEPSO at 2°C to 8°C (36°F to 46°F).
*Do not freeze.
 
{{Portal bar | Medicine}}
 
[[Category:Antisense RNA]]
[[Category:Muscular dystrophy]]
[[Category:Therapeutic gene modulation]]
[[Category:Orphan drugs]]
{{coststubd}}
{{pharma-stub}}

Revision as of 04:04, 13 February 2025

An article about the drug Viltolarsen


Viltolarsen
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Viltolarsen is a pharmaceutical drug used in the treatment of Duchenne muscular dystrophy (DMD), a severe type of muscular dystrophy characterized by rapid progression of muscle degeneration. Viltolarsen is specifically designed to target mutations in the dystrophin gene that are amenable to exon 53 skipping.

Mechanism of Action

Viltolarsen is an antisense oligonucleotide that works by binding to exon 53 of the dystrophin pre-mRNA. This binding causes the exon to be skipped during mRNA splicing, resulting in the production of a truncated but functional dystrophin protein. This mechanism is intended to partially restore the function of the dystrophin protein in patients with specific genetic mutations.

Clinical Use

Viltolarsen is indicated for the treatment of patients with Duchenne muscular dystrophy who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. It is administered via intravenous infusion and is typically given on a weekly basis.

Side Effects

Common side effects of Viltolarsen include injection site reactions, upper respiratory tract infections, and fever. Patients receiving Viltolarsen should be monitored for potential adverse reactions, and the drug should be used with caution in patients with renal impairment.

Development and Approval

Viltolarsen was developed by Nippon Shinyaku and NS Pharma. It received accelerated approval from the United States Food and Drug Administration (FDA) in 2020 based on its ability to increase dystrophin production in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Related pages

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