NHS strikes deal for treatment for rare genetic disease in babies

NHS England has made a commercial deal for sebelipase alfa, an enzyme-replacement therapy, which will become the first treatment available on the NHS for Wolman disease, a condition which presents in babies and children under two years old.

Wolman disease is a type of lysosomal acid lipase deficiency and occurs in around 1 in 350,000 births. The disease causes a build-up of fat in cells in the liver, heart, blood vessels, and digestive system.

Previously, there had been no treatment options for Wolman disease on the NHS. Standard care was palliative and limited to managing symptoms.

Sebelipase alfa is an enzyme replacement therapy, which works by replacing an enzyme missing in the body, alongside a restricted, low-fat diet. Treatment involves weekly intravenous infusions which can be given at home – some patients may also have a blood and marrow/stem cell transplant.

John Stewart, national director for specialised commissioning, NHS England said: “It’s fantastic news that babies born with Wolman disease will be able to benefit from this life-saving treatment on the NHS from today.

“This is just the latest example of patients benefiting from innovative medicines secured through NHS commercial deals, following shortly after a new five-year medicines agreement was reached with the pharmaceutical industry that will enable rapid patient access to the treatments of tomorrow.”