FDA

FDA approves first-in-class drug for familial chylomicronemia syndrome

December 26, 2024

Brand name: Tryngolza

Generic name: olezarsen

Manufacturer: Ionis

Approval date: December 19, 2024

FDA approved Tryngolza (olezarsen), an antisense oligonucleotide, as an adjunct to diet to reduce triglycerides (TG) in adults with familial chylomicronemia syndrome (FCS).

Efficacy

The efficacy and safety of Tryngolza were evaluated in the randomized, placebo-controlled, double-blind BALANCE trial (NCT04568434) involving 66 adult patients with FCS and fasting TG levels ≥880 mg/dL (average baseline TG level was approximately 2600 mg/dL). Primary endpoint was percent change in fasting TG levels from baseline to month 6 (average of weeks 23, 25, and 27) compared to placebo.

The average percent change in TG from baseline to month 6 in the Tryngolza group was -42.5% compared with the placebo group. Median percent and absolute changes in TG levels from baseline over time demonstrated a consistent lowering effect during the 12-month treatment period.

Safety

The most common adverse reactions in patients treated with Tryngolza were injection site reactions, decreased platelet count, and arthralgia.

Recommended dose

The recommended dosage of Tryngolza is 80 mg SC once monthly.

Sources:

FDA. (2024, December 19). FDA approves drug to reduce triglycerides in adult patients with familiar chylomicronemia syndrome. [Press release]. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-reduce-triglycerides-adult-patients-familial-chylomicronemia-syndrome

Ionis. (2024, December 19). Tryngolza (olezarsen) approved in U.S. as first-ever treatment for adults living with familial chylomiconemia syndrome as an adjunct to diet. [Press release]. https://www.multivu.com/ionis-pharmaceuticals/9295551-en-tryngolza-olezarsen-fda-approval

Ionis: Tryngolza (olezarsen). [Package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/218614s000lbl.pdf