Ionis Pharmaceuticals subsidiary Akcea Therapeutics has received a non-binding recommendation from the Division of Metabolism and Endocrinology Products Advisory Committee at the US Food and Drug Administration (FDA), for the approval of Waylivra to treat familial chylomicronemia syndrome (FCS).
The FDA will consider the committee’s opinion while reviewing the New Drug Application submitted by Akcea.
Formulated using Ionis’s antisense technology, Waylivra is designed to minimise the production of ApoC-III protein, which is found in the liver and associated with plasma triglycerides regulation.
Akcea Therapeutics CEO Paula Soteropoulos said: “The committee’s majority vote in favour of approval is an important positive step to bring Waylivra to people with FCS who have no adequate treatment options.
“We are committed to the FCS community and will continue to focus on bringing Waylivra, potentially the first and only treatment, to people living with this serious and potentially fatal disease.”
The committee’s decision is based on the results of two Phase III clinical trials and an open-label study that is currently underway.
In one trial. Waylivra achieved a statistically significant mean reduction in triglycerides of 77% from baseline and decreased risk of pancreatitis.
The most common adverse events observed during the trial were injection site reactions and platelet decline.
The drug is being studied in another Phase III study for familial partial lipodystrophy (FPL) patients. The data from this trial is expected to be reported next year.