NICE backs Sobi's Tryngolza for rare disease FCS

Patients in England and Wales with rare disease familial chylomicronaemia syndrome (FCS) will soon have a new treatment option, after Sobi's Tryngolza was recommended for NHS use by NICE.

In final draft guidance, the reimbursement authority has said that Tryngolza (olezarsen) can be prescribed for adults with genetically confirmed FCS who do not respond well enough to diet and conventional triglyceride-lowering treatments like statins and fibrate drugs.

Sobi's drug – which it licenses from Ionis - is the first alternative to Akcea's Waylivra (volanesorsen), which was recommended by NICE in 2020 for FCS patients at high risk of pancreatitis who have not responded well enough to diet and other triglyceride-lowering drugs.

The two drugs work in a similar way – both are antisense oligonucleotides targeting apoC-III – and would be offered to the same patient population, according to the guidance, which notes that the cost of Sobi's drug is "similar to or lower" than Waylivra.

They also have different dosing schedules, with Tryngolza given by injection once a month while Waylivra requires weekly shots for three months, and every two weeks thereafter. Meanwhile, Tryngolza also has a reduced risk of severe reductions in platelets, a recognised side effect of Waylivra that requires patients to have routine blood monitoring.

A group of rare genetic disorders, FCS affects somewhere between 110 and 120 people in the UK, although it is thought to be under-diagnosed. It causes very high levels of triglycerides in the blood.

People with FCS have to avoid fat in the diet as they either don't have the enzyme which breaks it down, or the enzyme they have doesn't work correctly. The disorder causes a range of symptoms including cognitive impairment, physical symptoms like pancreatitis, and poor mental health. In severe cases, it can lead to permanent organ damage and death.

"People living with FCS have extremely high triglyceride levels since childhood, which causes severe recurrent pain and unpredictable and potentially life-threatening episodes of acute pancreatitis," said Prof Handrean Soran, consultant physician and endocrinologist at Manchester University NHS Foundation Trust.

"Historically, we have had very limited treatments to manage this," he added. "Access to olezarsen through the NHS in England and Wales will represent an important step forward."

Sobi has exclusive rights to commercialise Tryngolza in all ex-US geographies, except Canada and China. The drug is still in the rollout phase in Europe, but is making upwards of $100 million a year in the US for Ionis.

Sharon Hall, general manager of Sobi UK, said the company is now "working with the NHS, clinical experts, and the wider FCS community to support the effective implementation of this recommendation and ensure eligible patients can benefit from this new treatment option."