Ionis has confirmed that the therapy will be available in the US before the end of 2024. Credit: Ionis Pharmaceuticals, Inc.

The US Food and Drug Administration (FDA) has approved Ionis Pharmaceuticals‘ TRYNGOLZA (olezarsen), designed as an adjunct to diet for reducing triglyceride levels in adults with familial chylomicronaemia syndrome (FCS), a genetic form of hypertriglyceridemia.

This is the first FDA-approved therapy that lowers triglyceride levels in adult patients. It offers a meaningful reduction in acute pancreatitis (AP) events when accompanied by a suitable low-fat diet. The self-administered treatment can be used once a month.

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The FDA’s decision was influenced by the outcomes from the double-blind, multicentre Phase III Balance trial.

This global, randomised, placebo-controlled study involved adult subjects with genetically confirmed FCS and fasting triglyceride levels of at least 880mg/dL. The therapy significantly reduced triglyceride levels and substantially lowered the incidence of AP events in the subjects.

Ionis Pharmaceuticals CEO Brett Monia stated: “Today’s FDA approval of TRYNGOLZA heralds the arrival of the first-ever FCS treatment in the US – a transformational moment for patients and their families. For the first time, adults with FCS can now access a treatment that substantially reduces triglycerides and the risk of debilitating and potentially life-threatening acute pancreatitis.

“The FDA approval of TRYNGOLZA is also a pivotal moment for Ionis, representing our evolution into a fully integrated commercial-stage biotechnology company – a goal we set out to achieve five years ago.”

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The company has confirmed that the therapy will be available in the US before the end of 2024.

TRYNGOLZA also received priority review from the US regulator and had previously been granted fast track, orphan drug and breakthrough therapy designations.

Olezarsen is under review in the European Union and has regulatory filings planned in other countries.

Currently, it is being evaluated in three Phase III clinical trials – CORE, CORE2, and ESSENCE – for the treatment of severe hypertriglyceridemia, although it has not yet been reviewed or approved for this indication by regulatory authorities.