FDA Advisory Committee Gives Green Light to Akcea Therapeutics Rare Disease Drug Waylivra

Green Light

Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals, is preparing for the possible launch of its therapeutic for the treatment of the rare lipid disorder familial chylomicronemia syndrome (FCS) following the thumbs up from a U.S. Food and Drug Administration advisory committee.

On Thursday the Endocrinologic and Metabolic Drugs Advisory Committee voted 12 to 8 in favor of volanesorsen, which will be marketed as Waylivra should it be approved. The FDA will provide a final ruling by Aug. 30. The advisory committee’s endorsement is not binding, but the FDA typically follows the recommendation of the panel experts. Waylivra is also under regulatory review in the European Union and Canada.

The FDA’s EMDA Committee voted in favor of Waylivra after reviewing data from two Phase III clinical trials. Data showed that FCS patients treated with Waylivra achieved a statistically significant mean reduction in triglycerides of 77 percent from baseline and decreased the risk of pancreatitis.

However, the eight dissenters were concerned over Waylivra’s safety profile – particularly the fact that the therapy caused dangerous drops in platelet counts in some patients. The most common adverse events in the Phase III studies were injection site reactions and platelet declines, Akcea said. When the company announced Phase III results in March 2017, it revealed that five volanesorsen-treated patients discontinued due to injection site reactions. While the platelet declines were typically well-treated, Akcea also revealed that five trial patients discontinued due to declines in platelets. The company also noted that there were three grade 4 thrombocytopenia reports in the trial. The three patients ceased taking the medication and the thrombocytopenia (low blood platelet count) ceased. It was these safety concerns that caused some of the FDA advisory committee members to reject Waylivra.  

Following the late Thursday announcement, shares of Akcea are up more than 16 percent in premarket trading. Parent company Ionis was up nearly 5 percent in early trading.

Paula Soteropoulos, chief executive officer of Akcea Therapeutics, said the majority vote in favor of Waylivra’s approval is “an important positive step” to bring the treatment to people with FCS “who have no adequate treatment options.”

“We look forward to working with the FDA to complete the final stages of regulatory review for Waylivra. 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,” Soteropoulos said in a statement.

FCS is a severe, rare disorder where patients have extremely high levels of triglycerides in their blood, causing a range of symptoms including potentially fatal attacks of acute pancreatitis. With limited LPL function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90 percent triglycerides. FCS patients are at risk of chronic complications due to permanent organ damage. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognitions that affect their ability to work.

Brett P. Monia, chief operating officer at Ionis, said Waylivra is the first drug to demonstrate substantial triglyceride lowering in clinical trials in people with FCS.

The drug is also currently in Phase III clinical development for the treatment of patients with familial partial lipodystrophy, or FPL. Akcea anticipates reporting top-line data from this study in 2019

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