Viltolarsen

What Is Viltolarsen?[edit | edit source]

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.

What are the uses of this medicine?[edit | edit source]

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.
This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO .
Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

How does this medicine work?[edit | edit source]

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.
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.

Who Should Not Use this medicine ?[edit | edit source]

This medicine have no usage limitations.

Is this medicine FDA approved?[edit | edit source]

It was approved for use in the United States in 2020.

How should this medicine be used?[edit | edit source]

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?[edit | edit source]

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?[edit | edit source]

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?[edit | edit source]

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?[edit | edit source]

In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

Overdose related information is also available online at poisonhelp.org/help.
In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?[edit | edit source]

There are no human or animal data available to assess the use of VILTEPSO during pregnancy.

Can this medicine be used in children?[edit | edit source]

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?[edit | edit source]

Store VILTEPSO at 2°C to 8°C (36°F to 46°F).
Do not freeze.

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