FDA Approves Ionis Pharmaceuticals' Rare Genetic Disease Therapy to Lower High-Level Of Fat In Blood

On Thursday, the FDA approved Ionis Pharmaceuticals, Inc.’s IONS Tryngolza (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a rare, genetic form of severe hypertriglyceridemia (SHTG) that can lead to acute pancreatitis (AP).

SHTG is a condition where the level of triglycerides (type of fat) in the blood is more than three times the normal level. 

Tryngolza is the first-ever FDA-approved treatment that significantly and substantially reduces triglyceride levels in adults with FCS and provides a clinically meaningful reduction in AP events when used with an appropriate diet (≤20 grams of fat per day).

Tryngolza is self-administered via an auto-injector once-monthly.

The FDA approval was based on Phase 3 Balance trial data in adult patients.

In the Balance study, Tryngolza 80 mg demonstrated a statistically significant placebo-adjusted mean reduction in triglyceride levels of 42.5% from baseline to six months (p=0.0084). 

Reductions from baseline to 12 months were further improved, with Tryngolza achieving a placebo-adjusted 57% mean triglyceride reduction. 

Tryngolza also demonstrated a substantial, clinically meaningful reduction in AP events over 12 months; one patient (5%) experienced one episode of AP in the Tryngolza group compared with seven patients (30%) who experienced 11 total episodes of AP in the placebo group. 

Tryngolza will be available in the U.S. before year end.

Olezarsen is currently being evaluated in three Phase 3 clinical trials – CORE, CORE2, and ESSENCE – for SHTG. Olezarsen has not been reviewed or approved for SHTG.

In November, Ionis Pharmaceuticals announced the pivotal Phase 3 study design following alignment with the FDA on ION582 for Angelman syndrome (AS). The trial is expected to start in the first half of 2025.

AS typically presents in infancy and is characterized by profound intellectual disability, impaired verbal abilities, and severe motor impairment.

Price Action: At last check on Friday, IONS stock was up 3.79% at $36.94 during the premarket session.

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