Long-term effects of volanesorsen on triglycerides and pancreatitis in patients with familial chylomicronaemia syndrome (FCS) in the UK Early Access to Medicines Scheme (EAMS).
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Author
Jones, AlanPeers, Katherine
Wierzbicki, Anthony S
Ramachandran, Radha
Mansfield, Michael
Dawson, Charlotte
Ochoa-Ferraro, Antonio
Soran, Handrean
Jenkinson, Fiona
McDowell, Ian
Downie, Paul
Hamilton, Paul
Jones, Richard D
Publication date
2023-05-15
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Background and aims: The VOL4002 study assessed the efficacy and safety of volanesorsen in 22 adults with genetically confirmed familial chylomicronaemia syndrome (FCS) treated in the UK Early Access to Medicines Scheme (EAMS), with ("prior exposure") or without ("treatment naive") previous treatment in the APPROACH and/or APPROACH-OLE volanesorsen phase 3 studies. Methods: Data collection focused on triglyceride (TG) levels, platelet counts and pancreatitis events. Pancreatitis incidence during volanesorsen treatment was compared against the 5-year period preceding volanesorsen exposure. Volanesorsen 285 mg was self-administered subcutaneously once every 2 weeks. Results: Individual patient volanesorsen exposure ranged from 6 to 51 months (total cumulative exposure, 589 months). Among treatment-naive patients (n = 12), volanesorsen treatment resulted in an averaged median 52% reduction (-10.6 mmol/L) from baseline (26.4 mmol/L) in TG levels at 3 months, which were maintained through time points over 15 months of treatment (47%-55% reductions). Similarly, prior-exposure patients (n = 10) experienced a 51% reduction (-17.8 mmol/L) from pre-treatment baseline (28.0 mmol/L), with reductions of 10%-38% over 21 months of treatment. A comparison of pancreatitis event rates found a 74% reduction from the 5-year period before (one event/2.8 years) and during (one event/11.0 years) volanesorsen treatment. Platelet declines were consistent with observations in phase 3 clinical trials. No patient recorded a platelet count <50 × 109/L. Conclusions: This longitudinal study supports the efficacy of volanesorsen in patients with FCS for lowering TG levels over treatment periods up to 51 months with no apparent safety signals related to increased duration of exposure.Citation
Jones A, Peers K, Wierzbicki AS, Ramachandran R, Mansfield M, Dawson C, Ochoa-Ferraro A, Soran H, Jenkinson F, McDowell I, Downie P, Hamilton P, Jones RD. Long-term effects of volanesorsen on triglycerides and pancreatitis in patients with familial chylomicronaemia syndrome (FCS) in the UK Early Access to Medicines Scheme (EAMS). Atherosclerosis. 2023 Jun;375:67-74. doi: 10.1016/j.atherosclerosis.2023.05.008. Epub 2023 May 15.Type
ArticleAdditional Links
https://www.sciencedirect.com/journal/atherosclerosisPMID
37253281Journal
AtherosclerosisPublisher
Elsevierae974a485f413a2113503eed53cd6c53
10.1016/j.atherosclerosis.2023.05.008