Waylivra

Waylivra is the brand name for volanesorsen, an antisense oligonucleotide drug developed to treat certain severe triglyceride disorders. It is designed to lower triglyceride levels by reducing the production of apolipoprotein C-III, a protein that regulates triglyceride-rich lipoproteins.

Volanesorsen works by binding to the messenger RNA for APOC3 in the liver, thereby decreasing the synthesis of ApoC-III. Reduced ApoC-III levels lead to decreased circulating triglycerides and a reduction in chylomicrons and very low-density lipoprotein (VLDL) particles. This mechanism targets a key driver of hypertriglyceridemia in affected patients.

Waylivra’s approved use is for adults with familial chylomicronemia syndrome (FCS) to lower triglyceride levels. It has also been studied for other forms of severe hypertriglyceridemia, but its availability and indications vary by country and regulatory status. The drug is administered by subcutaneous injection, with dosing schedules determined by regional labeling and clinician judgment. Patients typically require regular monitoring by healthcare professionals.

Safety concerns have been a major consideration in its use. The most notable issue is thrombocytopenia, which can increase bleeding risk, necessitating periodic monitoring of platelet counts. Other common adverse effects include injection-site reactions, fatigue, flu-like symptoms, and mild gastrointestinal or hematologic effects. As with any therapy, the risk-benefit profile should be evaluated by clinicians on an individual basis.

Regulatory status varies by region. In some jurisdictions, Waylivra has been approved with strict monitoring requirements; elsewhere development has faced safety-related challenges and non-approval decisions.