Familial Chylomicronemia Syndrome
Familial chylomicronemia syndrome (FCS) is a condition that is considered ultra-rare. It’s caused when the enzyme called lipoprotein lipase (LPL) is impaired. It is can result in pancreatitis, hypertriglyceridemia, pancreatogenic diabetes, abdominal pain, fatigue, and psychosocial complications. There are no treatments currently approved for the disease. Symptoms can be debilitating and the constant fear of pancreatitis can significantly affect patient’s quality of life.
There is a desperate need for more options for FCS patients and thankfully, one may be coming soon.
Trials for WAYLIVRA
Akcea and Ionis conducted two Phase 3 trials examining WAYLIVRA in FCS and it is the positive results from this trial that are setting the basis for their application for marketing approval. One of these trials, called COMPASS, was the largest study ever conducted for FCS. It showed WAYLIVRA was effective in reducing triglycerides by 77% in just three months of treatment.
WAYLIVRA is currently in a Phase 3 trial for Familial partial lipodystrophy (FPL) as well and they expect results from this trial by the middle of the year.
Progress Toward Approval
Akcea is working to get WAYLIVRA approved for FCS in the United States and Canada, but they are closest to approval in Europe. They are expecting approval to come in the next couple of months. Partly, this is due to the positive opinion of WAYLIVRA recently given to Akcea from the Committee for Medicinal Products for Human Use (CHMP), which is a part of the European Medicines Agency (EMA). The recommendation from this committee for the drugs conditional marketing authorization is a huge step on the way to its approval. Their positive opinion will now be sent to the European Commission (EC). The EC will then grant it marketing authorization. This authorization will be conditional until Akcea completes a post-authorization safety study. Akcea plans to complete this trial using a patient registry.
Specifically, this approval will be for patients with FCS who have not responded to diet and triglyceride lowering therapy and have a high risk for developing pancreatitis. WAYLIVRA will be used in conjunction with diet. This approval will span the entire European Union.
Stay tuned to hear more updates on this journey toward approval! In the meantime, you can read more about WAYLIVRA here.