Viltepso®

Understanding Viltepso® 

Viltepso® (viltolarsen) is an FDA-approved antisense oligonucleotide therapy designed to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene amenable to exon 53 skipping. By promoting exon 53 skipping during mRNA processing, Viltepso® facilitates the production of a truncated yet functional dystrophin protein, which is crucial for muscle fiber integrity. This therapy aims to slow the progression of muscle degeneration associated with DMD.

How Viltepso® Works:

• Exon Skipping Mechanism: Viltolarsen binds to specific sequences of the pre-mRNA, causing the cellular splicing machinery to skip exon 53 during mRNA processing. This results in the production of a shorter, functional dystrophin protein that can partially compensate for the missing or defective dystrophin in DMD patients.

• Administration: The recommended dosage is 80 mg/kg of body weight, administered once weekly as a 60-minute intravenous infusion by a healthcare professional. The dosage is calculated based on the patient’s weight to ensure appropriate therapeutic levels.

FDA Approval:

• Viltepso® received accelerated approval from the U.S. Food and Drug Administration (FDA) on August 12, 2020, for the treatment of DMD in patients amenable to exon 53 skipping. This approval was based on observed increases in dystrophin production in skeletal muscle. Continued approval may depend on verification of clinical benefits in confirmatory trials.

For more information, please visit the Viltepso® patient website and speak with your healthcare provider to determine if Viltepso® is the right treatment option for you.