Rare Daily Staff

A U.S. Food and Drug Administration Advisory Committee voted to recommend that the agency grant marketing approval to volanesorsen, Akcea Therapeutics experimental therapy for familial chylomicronemia syndrome, a rare enzyme deficiency that leads to potentially dangerous levels of fat in the blood.

FCS is a condition in which patients cannot metabolize triglyceride-rich lipid particles called chylomicrons due to a deficiency in the enzyme lipoprotein lipase. As a result, people with FCS have high levels of triglycerides in the blood, which leads to a range of symptoms including potentially fatal attacks of acute pancreatitis.

The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 12 to 8 in favor of approval of volanesorsen for the treatment of FCS.

The vote is a welcome relief for Akcea. There had been speculation that safety concerns about volanesorsen could derail the company’s efforts to win approval. The briefing document provided to the review committee noted while that despite the magnitude of the drug’s effect on fasting triglyceride levels, “the volanesorsen review team remains uncertain whether the benefits of volanesorsen outweighs its risks, considering safety concerns with this product.”

The reviewers expressed concern that the drug could cause a rapid and unpredictable reduction in platelets to extremely low levels that could result in serious bleeding.

The FDA set a PDUFA date of August 30, 2018 for its decision.

May 10, 2018