The U.S. Food and Drug Administration has approved Ionis Pharmaceuticals (NASDAQ: IONS) drug to treat a rare genetic disorder, making it the company’s first wholly-owned drug, the health regulator’s website showed on Thursday.
The drug, chemically known as olezarsen and branded as Tryngolza, is also the first approved treatment for familial chylomicronemia syndrome (FCS), a disorder that prevents the body from breaking down fats and is characterized by extremely high levels of triglycerides.
FCS is caused by the deficiency or impaired function of the lipoprotein lipase enzyme, responsible for breaking down triglycerides — which leads to inflammation of the pancreas when accumulated in the blood.
A strict low-fat diet is the current first-line treatment for the condition.
The drug was approved as an add-on treatment to diet to reduce triglycerides in adults with the condition.
The disorder affects fewer than 5,000 people in the U.S., according to government data.
“We don’t see any hurdles on supply. We have manufactured and (are) ready to go,” CEO Brett Monia told Reuters ahead of the approval.
“We will be planning to price olezarsen as you would, a first-time treatment for a severe, rare disease,” Monia said.
The company said it will announce details on the drug’s pricing in an investor conference call.
Ionis’ application for the drug was based on data from a late-stage study in which it helped reduce triglyceride levels by 44% in patients, compared to a placebo.
Patients are still required to follow recommended dietary restrictions, Monia said.
Given the low prevalence of the disease and the entry of rival drugs by the second half of next year, BMO Capital Markets analyst Kostas Biliouris does not expect FCS to be a significant opportunity for the company.
Biliouris estimates peak sales of $341 million in FCS.
The company has partnered with several major drugmakers such as Biogen (NASDAQ: BIIB) and Swiss-based Novartis (NYSE: NVS) to develop other treatments.
