Ionis Pharma, Sobi Get Positive CHMP Opinion For TRYNGOLZA In Familial Chylomicronemia Syndrome

(RTTNews) - Ionis Pharmaceuticals, Inc. (IONS) and Sobi Friday said that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion of TRYNGOLZA as an adjunct to diet in adult patients with familial chylomicronemia syndrome (FCS)

A decision on this is expected by the fourth quarter of this year.

FCS is a rare and genetic disorder characterized by extremely high levels of triglycerides, causing recurrent episodes of acute pancreatitis.

The CHMP opinion is based on positive data from the Phase 3 Balance study, which showed statistically significant reductions in triglyceride levels at six months in patients treated with TRYNGOLZA. This was maintained for up to 12 months. Additionally, TRYNGOLZA demonstrated a substantial and clinically meaningful reduction in acute pancreatitis events over 12 months.

The drug is already approved in the U.S. for adults with FCS.