While rare, familial chylomicronemia syndrome can be diagnosed at any time during a patient’s life, although it is present at birth and commonly diagnosed during childhood. The autosomal recessive disorder results in a notable impact on patient quality of life, primarily through low-fat dietary restriction and complications, including chronic abdominal pain and recurrent pancreatitis.
Today, we are taking a real-time look at the latest developments regarding familial chylomicronemia syndrome in relation to clinical guidance, FDA approvals, and ongoing clinical trials.
Current Familial Chylomicronemia Syndrome Clinical Guidance
- Familial Chylomicronemia Syndrome
- National Lipid Association (NLA)
- March 2025
Recent FDA Approvals for Familial Chylomicronemia Syndrome
Below are the recent medications and therapies recently approved by the FDA that we anticipated being incorporated into future clinical guidelines:
- Redemplo (plozasiran)
- Arrowhead Pharmaceuticals
- Approved: November 2025
- Redemplo was approved as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).
- Tryngolza (olezarsen)
- Ionis Pharmaceuticals
- Approved: December 2024
- Tryngolza was approved as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).
Ongoing, Phase 3 Clinical Trials for Familial Chylomicroemia Syndrome
The following are ongoing, Phase 3 clinical trials based in the United States that we anticipate may impact future familial chylomicronemia syndrome clinical guidelines.
- Ionis Pharmaceuticals
- Anticipated Primary Completion: June 2026
- Ionis Pharmaceuticals
- Anticipated Primary Completion: February 2028
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