Concept image for the rare genetic condition known as Wolman disease

Landmark NHS deal signals first ever treatment offered for Wolman disease

The NHS has broken new ground today as a first-ever treatment for a rare genetic condition that affects babies and children under the age of two has been approved.

The National Institute for Health and Care Excellence’s (NICE) final draft guidance recommending sebelipase alfa – marketed as Kanuma – follows a commercial deal struck by NHS England to benefit Wolman disease patients.

Wolman disease is a type of lysosomal acid lipase deficiency that occurs in around one in 350,000 births, which can cause a variety of symptoms including enlarged liver and spleen, jaundice and anaemia.

Previously, the NHS offered no treatment for Wolman disease with standard care being palliative and limited to managing symptoms – NICE’s recommendation will see the life-saving medicine fast-tracked to eligible patients immediately via NHS England’s Innovative Medicines Fund.

                                                                      Video credit: Canva

In conjunction with a low-fat diet, sebelipase alfa works as an enzyme replacement therapy with treatment involving weekly intravenous infusions.

The therapy is manufactured by Alexion and will be delivered at:

Professor Simon Jones, who is the clinical director of the NIHR Manchester Clinical Research Facility, where clinical trials for sebelipase alfa were conducted, described today as a “major milestone” in the treatment of infants with Wolman disease.

He said: “I am thrilled to see that this lifesaving drug will now be available on the NHS as a specialist service for the benefit of more children and families with this rare genetic condition.

“More than a decade on since our world-first clinical trials, I am incredibly proud of what our research and clinical teams here in Manchester have delivered collaboratively, which has contributed to this successful outcome.”

NHS England’s chief executive, Amanda Pritchard, added: “I am delighted the NHS can now, for the first time, offer a life-changing treatment to families facing this enormously difficult condition.”

Image credit: iStock

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NHE May/June 2024

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