|Drug class||Antisense oligonucleotide|
|Main uses||Duchenne muscular dystrophy (DMD)|
|Side effects||upper respiratory tract infection, injection site reaction, cough, fever|
|Typical dose||80 ug/kg|
|Chemical and physical data|
|Molar mass||6924.910 g·mol−1|
Viltolarsen, sold under the brand name Viltepso, is a medication used to treat certain cases of Duchenne muscular dystrophy (DMD). Specifically it is used for the approximately 8% of people with DMD who have a mutation that is amenable to exon 53 skipping. As of 2020 it was unclear if it results in improvements. It is given by injection into a vein over an hour.
Common side effects include upper respiratory tract infection, injection site reaction, cough, and fever. Other potential concerns include kidney problems. It is a morpholino antisense oligonucleotide.
Viltolarsen was approved for medical use in the United States in 2020. It is not approved in Europe, as of 2020, though has orphan designation. In the United States it costs about 1,500 USD per 250 mg as of 2021. This is about 730,000 USD for a person who is 30 kg.
While it results in improvements in biochemical markers evidence as of 2020 does not support improved outcomes for the people affected.
The typical dose is 80 ug/kg once per week.
Although kidney toxicity was not observed in the clinical studies, the clinical experience is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides.
Viltolarsen was evaluated in two clinical studies with a total of 32 participants, all of whom were male and had genetically confirmed DMD. The increase in dystrophin production was established in one of those two studies, a study that included sixteen DMD participants, with eight participants receiving viltolarsen at the recommended dose. In the study, dystrophin levels increased, on average, from 0.6% of normal at baseline to 5.9% of normal at week 25. Trial 1 provided data for evaluation of the benefits of viltolarsen. The combined populations from both trials provided data for evaluation of the side effects of viltolarsen. Trial 1 was conducted at six sites in the United States and Canada and Trial 2 was conducted at five sites in Japan. All participants in both trials were on a stable dose of corticosteroids for at least three months before entering the trials.
The U.S. Food and Drug Administration (FDA) concluded that the applicant's data demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in people with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping. A clinical benefit of the drug has not been established. In making this decision, the FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease, and the lack of available therapies.
Society and culture
Viltepso is over $733,000 a year for a 30 kilogram (66 pound) patient.
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