CHMP Recommends First Treatment of FCS for Marketing Approval

The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion that an orphan drug, volanesorsen (Waylivra), receive a conditional marketing authorization. The drug would be the first medication approved for the treatment of the familial chylomicronemia syndrome (FCS).

FCS is a rare genetic disease that prevents the body from breaking down lipids; therefore, patients with this condition have extraordinarily high levels of triglycerides in their blood. Having such high levels of triglycerides can cause adverse events such as severe abdominal pain, potentially fatal attacks of acute pancreatitis, and memory loss.

“This positive CHMP opinion is an important step forward as we work to bring Waylivra to people living with FCS who currently have no treatment options. Once approved, Waylivra will be the first and only therapy for people living with the devastating challenges of FCS. We are now anticipating approval in Europe in the coming months,” Paula Soteropoulos, chief executive officer of Akcea Therapeutics, said in a statement.

The marketing application was submitted based on results from the phase 3 APPROACH study and the ongoing Approach Open Label Extension study. Results from the APPROACH trial, which enrolled 66 patients, making it the largest trial conducted in patients with FCS to date, found that treatment with Waylivra reduced triglycerides by 77% after 3 months compared with an increase of 18% in the placebo arm. The most common adverse events in the APPROACH trial were injection site reactions and reductions in platelet levels.

“The FCS community is encouraged by the positive CHMP opinion and we remain very hopeful that people living with FCS will soon have an approved treatment available in the [European Union]. Without a treatment, people living with FCS have debilitating daily symptoms as well as the constant fear of pancreatitis,” said Jill Prawer, founder and chair of the LPLD Alliance. “This is a positive step for people living with FCS who currently have no treatment options.”

The positive opinion will now be sent to the European Commission to make a final decision on a European Union-wide marketing authorization.