Viltepso

About Viltolarsen or Viltepso

• Viltolarsen is used to treat Duchenne Muscular Dystrophy (DMD) in patients with confirmed mutation of DMD gene that is amenable to exon 53 skipping.
• Exon 53 skipping is a type of gene therapy that uses antisense oligonucleotides to block the production of dystrophin protein that lacks exon 53, which allows the body to produce a shorter dystrophin protein that is still functional.
• Based on the rise in dystrophin production in skeletal muscle shown in Viltolarsen-treated patients, this indication has been given accelerated approval.
• The confirmation and description of clinical benefit in a confirmatory trial may be necessary in order for this indication to continue receiving approval.
• Patients are selected based on their medical history, diagnosis, and the recommendation of their healthcare provider to incorporate Viltolarsen into their condition management plan.

Strength:

Injection: IV infusion of 250 mg/5 mL (50 mg/mL) in a single-dose vial.

Recommended Dosage:

The drug is administered by a health care professional once a week directly into the bloodstream through intravenous (a vein) or IV infusion. It takes approximately an hour for Viltespo to be infused into the body. Before beginning the treatment, it is advisable to assess the levels of serum cystatin C, urine protein-to-creatinine ratio, and urine dipstick.

• A weekly dose of 80 mg/kg of body weight is recommended for. As an intravenous infusion, it takes 60 minutes to administer.
• If the needed Viltolarsen volume is below 100 mL, it must be diluted with 0.9% Sodium Chloride Injection, USP.
• Patients may need to adjust the dosage of medication according to their response and side effects.
• The recommended dosage of Viltolarsen for your individual needs should be discussed with a healthcare professional.

Important:

Patients must consult their treating doctor in case of experiencing any severe side effects, such as allergic reactions. It is a treatment that can help to slow the progression of DMD, Viltolarsen is a lifelong treatment. This means patients need to continue taking the medicine for as long as they get benefits from its effects. Do not discontinue the therapy unless your doctor advises it. This medication is not a substitute for standard care. Patients who are all taking Viltolarsen should continue to receive all the recommended treatments such as physical therapy and occupational therapy. If you have any concerns about it, please ask your healthcare professional.

Warnings & Precautions

• Viltolarsen has been associated with kidney toxicity in animal studies, and although clinical studies didn’t show kidney toxicity.
• Limited clinical experience exists, and kidney issues have been observed with some antisense oligonucleotides. Therefore, kidney function should be monitored in patients using Viltolarsen, especially in patients with pre-existing kidney disease.
• Hypersensitivity reactions, including anaphylaxis, have been reported, necessitating close monitoring and immediate discontinuation if such reactions occur.
• Viltolarsen is not recommended for pregnant women, and its presence in human milk is unknown, making it unsuitable for breastfeeding women.
• Viltolarsen should be used with caution in patients with liver disease, as there is an increased risk of liver toxicity in these patients.
• Viltolarsen should be used with caution in patients taking other medications that may interact with the current medicine.

Common Viltolarsen Side effects

• Cold and an upper respiratory infection
• Reaction at the injection site (redness, swelling, discomfort)
• Cough
• Fever
• Bruising
• Hives
• Joints pains
• Diarrhea
• Vomiting
• abdominal pain
• Fatigue
• Breathing difficulty
• Rash

Use in Specific Population:

People with all ages can use to get treated, including pediatric patients, and old people. Viltolarsen may be exposed to more people because it is primarily excrete unchanged in the urine. No precise dosage change may be advised for DMD patients with renal impairment. This is because the impact of lower skeletal muscle mass on creatinine values in DMD patients can make it challenging to determine appropriate dosage adjustments based solely on estimated glomerular filtration rate.

Storage and Handle:

Viltolarsen should be handled with care. Avoid contact with the skin, eyes, and mucous membranes. If accidental contact occurs, rinse the area with water immediately.

• Vial should be protected from light
• Do not Freeze the Viltolarsen
• Store at 2 degree to 8 degree