Position statement of the D-A-CH Society for Prevention of Cardiovascular Diseases on benefit-risk ratio of volanesorsen treatment in familial chylomicronemia syndrome (FCS)
Considering the ongoing consultations with EMA regarding the Committee for Medicinal Products for Human Use (CHMP) opinion for volanesorsen for the treatment of familial chylomicronemia syndrome (FCS), the executive and scientific board of the D-A-CH Society for Prevention of Cardiovascular Diseases (Germany, Austria, Switzerland, www.dach-praevention.eu) has evaluated the currently available data on the use of volanesorsen in FCS with other German experts in the field of severe and rare lipid disorders.
The D-A-CH Society for Prevention of Cardiovascular Diseases has been founded in 2012. It links specialists in the fields of lipid metabolism, dyslipidemias, diabetes mellitus, hypertension, lifestyle and nutrition and is a voice of jointly formulated strategies for primary, secondary and tertiary prevention of cardiovascular diseases. Two of our members have been involved as study centers for volanesorsen in FCS. As a scientific society, we are concerned about the complete response letter of the FDA for volanesorsen in FCS, because it is putting the US approval process on hold, for the time being.
As a group of specialized physicians, who see and treat FCS patients on a regular basis, we consider the benefit-risk ratio of treatment with volanesorsen for a firmly and unequivocally diagnosed FCS patient clearly on the positive side.
Without doubt there is a high unmet medical need for patients who suffer from clinical symptoms of their disease. In many cases, the burden of FCS has destroyed the social life of the patients, who lose their jobs, spouses, social relationships and ability to care for themselves independently.
So far there are no effective therapies for FCS, with treatment consisting only of a very inconvenient extreme low-fat diet, which is still often ineffective in preventing chylomicronemia and acute pancreatitis and their later complications such as diabetes mellitus type 3c or even worse.1
Receiving treatment with volanesorsen will meet this medical need, by lowering triglyceride levels in FCS patients below the threshold for triglyceride-induced acute pancreatitis in the majority of patients.2
Regarding the obvious risk of thrombocytopenia with volanesorsen, we are very confident that specialists who are experienced in the therapy of lipid disorders and are usually working in highly specialized clinical centers, are very well capable to handle such risk, especially with the use of an effective risk management plan, including a fixed dose reduction scheme, triggered by pre-defined levels of platelet count.
Therefore, we consider the overall benefit-risk ratio of volanesorsen in the treatment of FCS clearly positive and express the hope for future availability of this innovative treatment to patients and their treating specialist physicians.
Univ. Prof. Dr. med. Winfried März
on behalf of the executive and scientific board of the D-A-CH Society for Prevention of Cardiovascular Diseases
1. Chait A, Brunzell JD. Chylomicronemia syndrome. Advances in internal medicine 1992; 37:249-73.
2. Blom DJ, Gaudet D. Treatment with volanesorsen reduced triglycerides and pancreatitis in patients with familial chylomicronemia syndrome and severe hypertriglyceridemia vs placebo: results of the APPROACH and COMPASS studies. Poster presented at the EPC 2018; Berlin, Germany.