Viltepso (Viltolarsen) for Duchenne Muscular Dystrophy

Introduction

Duchenne Muscular Dystrophy (DMD) is a rare and debilitating genetic disorder that affects young boys, gradually robbing them of their muscle strength and independence. While there’s no cure for DMD, recent breakthroughs in medical research have led to the development of promising treatments, including Viltepso (Viltolarsen). This comprehensive guide will explore Viltepso’s uses, dosage, potential side effects, interactions, and essential warnings. We’ll also touch on recent research and advancements in this critical area of medicine.

Viltepso: An Innovative Treatment

The FDA approved Viltepso (viltolarsen) on August 12, 2020 [1]. Viltepso, an active ingredient called Viltolarsen, is an antisense oligonucleotide designed to address Duchenne muscular dystrophy (DMD) in individuals with a verified genetic mutation in the DMD gene. Administered through intravenous infusion, Viltepso presents a clear and colorless solution. This treatment has shown promise in increasing dystrophin levels, a crucial protein for muscle health in DMD patients.

Uses

VILTEPSO, an antisense oligonucleotide, is prescribed for managing Duchenne muscular dystrophy (DMD) in individuals with a confirmed DMD gene mutation that can benefit from exon 53 skipping. DMD is a rare inherited disorder that causes progressive muscle weakness and degeneration. 

Mechanism 

Viltepso is formulated to target exon 53 of dystrophin pre-mRNA, aiming to induce the exclusion of this specific exon during mRNA processing. This process is intended for patients with genetic mutations that can benefit from exon 53 skipping [2]. Exon 53 skipping is designed to facilitate the generation of an internally truncated dystrophin protein in individuals with genetic mutations that are amenable to this exon skipping approach.

Dosage and Administration

To begin Viltepso injection, essential assessments include serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio measurements. The recommended dosage is 80mg/kg via a 60-minute intravenous infusion. If the required Viltepso volume is under 100mL, dilute it in 0.9% Sodium Chloride Injection, USP. Viltepso injection is available in single-dose vials at a concentration of 250mg/5mL (50mg/mL). These steps ensure the safe and effective use of Viltepso for Duchenne Muscular Dystrophy.

Side Effects

Common adverse reactions Viltepso users report include upper respiratory tract infections, injection site reactions, cough, and fever. While kidney toxicity has been observed in animals receiving Viltepso, it was not detected in clinical studies. However, monitoring kidney function is advisable. Any persistent increase in serum cystatin C or proteinuria should prompt consultation with a pediatric nephrologist.

Interactions

Viltepso has not been studied in pregnant or breastfeeding women. Therefore, if you are planning to become pregnant, breastfeeding, or considering breastfeeding, it’s essential to discuss the potential risks with your healthcare provider. Additionally, there is limited information regarding Viltepso use in patients with hepatic impairment.

Warnings and Precautions

Although clinical studies have not detected kidney toxicity in Viltepso users, it is worth noting that it has been observed in animals receiving the treatment. As such, kidney function should be closely monitored during Viltepso treatment [3]. A pediatric nephrologist should promptly evaluate any signs of persistent increases in serum cystatin C or proteinuria.

Conclusion

Viltepso (Viltolarsen) is a promising treatment option for patients with DMD with its confirmed gene mutation. While Viltepso shows great promise, patients should consider the associated warnings, precautions, and potential drug interactions. It is crucial to consult with a healthcare professional to thoroughly evaluate the risks and benefits of Viltepso in the context of individualized treatment plans. The ongoing research in this field holds the potential for further advancements, providing new avenues of hope for those affected by this rare genetic disorder. Viltepso price in India depends on the product requirement. Request more details by contacting our Patient Support Team at ‎(+91) 9315705373 or help@sansfro.com. 

Reference:

1. https://www.drugs.com/history/viltepso.html  
2. https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/212154s000lbl.pdf 
3. https://www.prnewswire.com/news-releases/ns-pharma-reports-on-results-of-viltepso-viltolarsen-injection-after-four-years-of-treatment-in-open-label-extension-trial-in-duchenne-muscular-dystrophy-301656722.html