Akcea has been granted a conditional approval for Waylivra, its antisense drug for rare inherited disorder familial chylomicronemia syndrome (FCS), in Europe.
The European Commission has cleared Waylivra (volanesorsen) as an adjunct to diet in adult patients with confirmed FCS who are at high risk for pancreatitis and have not responded well enough to diet and triglyceride lowering therapy. The EU is the first market for the new drug.
It’s a big win for Akcea, a subsidiary of Ionis, as the US FDA turned down its marketing application for Waylivra last August despite a 12 to 8 vote in favour of approval from its own advisory committee a year ago. The FDA had voiced concerns about the drug’s safety, particularly some reductions in blood platelet counts (thrombocytopenia) among patients treated with the antisense agent.
FCS is a rare genetic disease that prevents the body from breaking down fats, and results in extremely high levels of triglycerides in the blood. This causes a range of symptoms including severe abdominal pain, potentially fatal attacks of acute pancreatitis, hepatosplenomegaly, diabetes, lack of concentration, memory loss and fat-filled spots on the skin.
Currently, the only option for patients is a severely restricted low fat diet of 10-20 grams of fat a day which often fails to remove the threat of pancreatitis.
Waylivra will be Akcea’s second drug to reach the market in a year after transthyretin-related amyloidosis (ATTR) therapy Tegsedi (inotersen), which competes head-to-head with Alnylam’s rival Onpattro (patisiran) product.
The new drug will launch into a market with no rivals as it is the only approved treatment for FCS, with its European debut scheduled to take place in Germany before the end of the year with additional launches in 2020.
“Waylivra is the only treatment available for patients with FCS which makes this approval a landmark event for the global FCS community,” commented Jules Payne, chair of patient organisation FH Europe which represents FCS patients.
“Patients, their caretakers and their families have been suffering without any therapeutic option,” she said. “Now, patients across Europe can access a medicine that may help address their severely elevated triglycerides which can give them hope for better health.”
The EU approval has also been hailed by the FCS Foundation in the US, which called once again for the FDA to find a way towards making the drug available to US patients with the rare disease.
The organisation held a teleconference with the US regulator in March, asking the FDA to take a similar approach to the EU in granting a approval with “requirements in monitoring and safety” to offset any concerns about its clinical profile.
In Europe, Akcea has committed to a non-interventional post-authorisation safety study (PASS), based on a patient registry, that will continue to generate data on its safety and efficacy.
It is estimated that there are between 3,000 to 5,000 people living with FCS worldwide, with approximately 1,000 people living with FCS in Europe.