Reason for request
- Key points
Favourable opinion for reimbursement only in patients with genetically confirmed familial chylomicronemia syndrome (FCS), in whom response to diet and triglyceride lowering therapy has been inadequate, and with a history of pancreatitis.
- What therapeutic improvement?
Slight therapeutic improvement in the current treatment of FCS.
- Role in the care pathwayWhat role for the product ?
The treatment of familial chylomicronemia syndrome is currently based on a life-long low-fat diet with 15 to 20 g of fat per day. The aim of this diet is to maintain triglyceride levels < 1000 mg/dL, in particular to limit the risk of occurrence of acute pancreatitis.
The fibrates and omega-3 used in other dyslipidemias have a marginal efficacy in patients with familial chylomicronemia syndrome.
Role of the medicinal product in the care pathway
WAYLIVRA is a first-line treatment in patients with genetically confirmed familial chylomicronemia syndrome (FCS), with a history of acute pancreatitis.
In patients with no history of acute pancreatitis, WAYLIVRA has no place in the care pathway.
- Special recommendations
The decision to initiate WAYLIVRA treatment should be taken during multidisciplinary review meetings within a rare pancreatic diseases reference centre (PaRaDis) or, otherwise, a multidisciplinary review meeting organised by the NSFA (New French Atherosclerosis Society) given the specific population likely to benefit from this treatment in view of the efficacy data and safety profile, particularly with respect to the risk of thrombocytopenia (the treatment is accompanied by regular platelet count monitoring).
the clinical benefit of WAYLIVRA is:
Clinical Added Value
WAYLIVRA (olanesorsen) provides a minor clinical added value (CAV IV) in the current treatment of FCS.