Expert: GalNAc-conjugated antisense oligonucleotide (ASO) targeting hepatic APOC3 mRNA. ApoC-III inhibits lipoprotein lipase (LPL) activity and hepatic uptake of triglyceride-rich lipoproteins. Reduction of ApoC-III restores triglyceride clearance via both LPL-dependent and LPL-independent pathways, which is critical for FCS patients who have genetic LPL deficiency.

Everyday: A gene-silencing drug that blocks the liver from making a protein (ApoC-III) that slows down fat clearance from blood. ApoC-III is like a traffic cop that stops fat particles from being removed — silencing it lets the body clear triglycerides much faster. Given as a monthly injection under the skin.

Targets: []

Primary EP: [{"id":"appr-ep1","name":"% triglyceride reduction at 3 months","type":"PRIMARY","unit":"%","notes":"Placebo-adjusted difference: ~95 percentage points. Absolute mean TG decrease: -1,712 mg/dL in vola

Safety: Thrombocytopenia (platelets <140K): 76% volanesorsen vs 24% placebo. Grade 4 thrombocytopenia (<25K): 6% (2/33). Injection site reactions 79% (mostly mild). 27% (9/33) discontinued due to AEs: 5 for p

Primary EP: [{"id":"bal-ep1","name":"Placebo-adjusted mean TG reduction at 6 months","type":"PRIMARY","unit":"percentage points","p_value":"<0.001 (80mg); 0.08 (50mg, NS)","results":[{"ci":"-69.1 to -17.9","arm":

Safety: Favorable safety profile. Injection site reactions ~19% (mild). Mild platelet count reductions ~12% (NO cases <50K - key differentiator vs volanesorsen which had 76% thrombocytopenia). Arthralgia ~9%.

Tryngolza - Severe Hypertriglyceridemia - EMA Submission 2026

GalNAc-conjugated ApoC-III ASO for FCS. FDA approved Dec 2024. EU launch initiated early 2026. Phase 3 ongoing for broader severe hypertriglyceridemia (sHTG) indication — much larger market. Licensed from Ionis Pharmaceuticals. SOBI also markets Waylivra (volanesorsen, first-gen ApoC-III ASO) but Tryngolza is the successor with better safety (no thrombocytopenia) and monthly dosing.