Volanesorsen

Volanesorsen is an antisense oligonucleotide that is designed to reduce the production of Apolipoprotein C-III (ApoC-III), a protein produced in the liver that plays a central role in the regulation of plasma triglycerides.

Mechanism of Action

Volanesorsen works by inhibiting the production of ApoC-III, a protein that regulates the breakdown of triglycerides. High levels of ApoC-III can lead to the accumulation of triglycerides in the blood, a condition known as hypertriglyceridemia. By reducing the levels of ApoC-III, volanesorsen can help lower the levels of triglycerides in the blood.

Clinical Trials

Volanesorsen has been studied in several clinical trials for the treatment of familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL). In these trials, volanesorsen was shown to significantly reduce triglyceride levels in patients with these conditions.

Side Effects

The most common side effects of volanesorsen include injection site reactions, flu-like symptoms, and a decrease in platelet count. In some cases, these side effects can be serious and may require medical attention.

Approval Status

Volanesorsen has been approved by the European Medicines Agency (EMA) for the treatment of adult patients with FCS. It is also under review by the Food and Drug Administration (FDA) in the United States.

See Also

• Antisense therapy
• Hypertriglyceridemia
• Familial chylomicronemia syndrome
• Familial partial lipodystrophy

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