NICE Backs Sobi’s Tryngolza for Adults with Rare Genetic Triglyceride Disorder

Rare Daily Staff

England’s health technology value watchdog has issued final draft guidance recommending Sobi’s Tryngolza (olezarsen) as a National Health Service treatment option for adults with genetically confirmed familial chylomicronaemia syndrome, a rare inherited condition that can cause dangerously high blood-fat levels and recurrent pancreatitis.

The National Institute for Health and Care Excellence (NICE) recommends the drug only for people whose condition hasn’t responded adequately to a very low-fat diet and conventional triglyceride-lowering medicines, such as statins or fibrates. Once guidance is final, NHS England must make the drug available within 30 days of publication when clinicians consider it the most suitable option.

FCS is a genetic disorder in which the body cannot properly clear triglycerides, a type of fat, from the bloodstream. Patients may need to follow an extremely restrictive low-fat diet. They can also face severe abdominal pain and potentially life-threatening inflammation of the pancreas.

For patients whose triglyceride levels stay poorly controlled, the usual treatment is Waylivra (volanesorsen). Like Tryngolza, it targets apolipoprotein C-III (APOC-3). NICE said Tryngolza works in a similar way and would be used at the same point in care.

NICE concluded that Tryngolza provides enough benefit and value for money to be used routinely across the NHS for this group. That depends on a confidential commercial discount negotiated with the manufacturer.

Clinical-trial data showed that Tryngolza cut triglyceride levels more effectively than placebo. The trial did not compare it directly with Waylivra.

Indirect comparisons suggested the two drugs may reduce fasting triglycerides by similar amounts. NICE called those results uncertain and said they can’t show whether one drug works better than the other. The committee still judged the clinical effectiveness likely to be similar, because the drugs act in similar ways. Clinical experts supported that view.

NICE also noted practical advantages for Tryngolza. It is expected to be better tolerated, needs fewer injections and doesn’t require the routine platelet monitoring that Waylivra does.

Tryngolza’s list price is about $32,800 (£24,772) per 80-mg pre-filled pen, excluding VAT. The NHS will pay less through a patient access scheme, and the size of the discount is confidential. NICE said the drug’s costs are similar to or lower than those of Waylivra.

Patients already taking Tryngolza through the NHS before the final guidance is published can continue under their existing funding. They can stay on it until they and their NHS clinician decide it is appropriate to stop.

NHS Wales generally has 60 days from the first publication of final draft guidance to fund a NICE-recommended treatment.