Waylivra (volanesorsen) is a selective P2X3 receptor antagonist. It is indicated for the treatment of adult patients with genetically confirmed familial chylomicronemia syndrome (FCS), particularly those who have a poor response to diet and triglyceride-lowering therapy and are at high risk of pancreatitis.

Side Effects of Waylivra (Volanesorsen)

Injection Site Reactions

Erythema (redness) at the injection site.

Pain at the injection site.

Swelling at the injection site.

Discoloration, induration, and pruritus at the injection site.

Bruising, bleeding, and numbness at the injection site.

Hematological System-Related Side Effects

The incidence of decreased platelet count is 29%.

The incidence of thrombocytopenia is 21%.

Leukopenia and lymphopenia.

Eosinophilia.

Immune thrombocytopenic purpura.

Systemic Side Effects

Headache.

Myalgia.

Chills.

Fatigue and lassitude.

Fever.

Severe Side Effects of Waylivra (Volanesorsen)

Risk of Renal Toxicity

Proteinuria ≥ 500 mg/24 hours.

Serum creatinine increase ≥ 0.3 mg/dL (26.5 μmol/L) and above the upper limit of normal.

Estimated creatinine clearance ≤ 30 mL/min/1.73 m² calculated by the CKD-EPI equation.

Risk of Hepatic Toxicity

Isolated increase in ALT or AST > 8 times the upper limit of normal.

Increase in ALT or AST > 5 times the upper limit of normal, persisting for ≥ 2 weeks.

Mild increase in ALT or AST accompanied by total bilirubin > 2 times the upper limit of normal or INR > 1.5.

Patients should immediately report any signs of bleeding.

Precautions for Waylivra (Volanesorsen) Administration

Platelet Monitoring and Dosage Adjustment

Platelet count must be measured before the start of treatment.

Platelet levels must be monitored at least every two weeks during treatment.

Adjust the treatment frequency and monitoring interval based on platelet levels.

Correct Injection Method

For subcutaneous use only; intramuscular or intravenous injection is not allowed.

Each pre-filled syringe is for single use only.

Allow the syringe to stand at room temperature for at least 30 minutes before injection.

Do not use other heating methods.

Injection sites include the abdomen, upper thigh area, or lateral upper arm area.

Storage Conditions

Store in a refrigerator at 2°C - 8°C.

Can be stored at room temperature (below 30°C) for up to 6 weeks.

Avoid injecting into areas with tattoos, moles, birthmarks, bruises, rashes, or inflamed skin.

Laboratory Monitoring Requirements

Conduct regular urine protein tests (once every quarter).

Evaluate serum liver enzymes and bilirubin every quarter.

Evaluate inflammation through erythrocyte sedimentation rate (ESR) every quarter.