US-based biotechnology company Ionis Pharmaceuticals has received the US Food and Drug Administration (FDA) approval for Tryngolza (olezarsen) to treat a rare genetic disease.
Tryngolza is an RNA-targeted medicine designed to reduce the body’s synthesis of apoC-III, a protein produced in the liver that is a key regulator of triglyceride metabolism.
The drug significantly reduces triglyceride levels in adults with FCS and offers a clinically meaningful reduction in AP events when used with an appropriate diet.
Tryngolza is indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a severe form of hypertriglyceridemia (sHTG).
FCS is a rare, genetic disease characterised by extremely elevated triglyceride levels, caused by impaired function of the enzyme lipoprotein lipase (LPL).
The US health regulator approved Tryngolza under its Priority Review, after granting Fast Track designation and Orphan Drug designation and Breakthrough Therapy designation.
Ionis CEO Brett Monia said: “We are proud of our long-standing partnership with the FCS community and are grateful to the patients, families and investigators who participated in our clinical studies, enabling Ionis to make this new treatment a reality.
“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.
“With our rich pipeline of potentially life-changing medicines, we expect Tryngolza to be the first in a steady cadence of innovative medicines we will deliver independently to people living with serious diseases.”
The FDA approval was supported by positive data from Balance, a global, multicentre, randomised, placebo-controlled, double-blind Phase 3 clinical trial in adults with FCS.
In the Phase 3 study, Tryngolza 80mg showed a statistically significant placebo-adjusted mean reduction in triglyceride levels of 42.5% from baseline to six months.
Also, Tryngolza achieved a placebo-adjusted 57% mean reduction in triglycerides from baseline to 12 months.
The drug showed a favourable safety profile, with injection site reactions, decreased platelet count and arthralgia as the most common adverse reactions.
Balance trial investigator Alan Brown said: “With no treatment options previously available, we were limited to relying only on extremely strict diet and lifestyle changes as the sole preventative treatment option.
“The FDA approval of TRYNGOLZA is an important moment for people living with FCS, their families and physicians who now, for the first time, have a treatment that significantly lowers triglycerides and decreases the risk of potentially life-threatening acute pancreatitis events, as an adjunct to a low-fat diet.
“I am excited to have a medicine I can prescribe to my patients that has been shown to change the course of their disease.”
Furthermore, Ionis is launching the Ionis Every Step initiative to provide patients and healthcare providers with access to services throughout the treatment journey.
Ionis Every Step will provide insurance and affordability support, along with services and resources, such as disease and nutrition education.
