Gene therapy will save babies at a record cost

Monday March 8th 2021

A powerful new gene therapy is to be used in the NHS to treat babies with spinal muscular atrophy, making it the most expensive therapy ever to gain official approval, it was announced today.

Each treatment will cost £1.79 million – enabling the service to rescue about 65 children and their parents annually from a life-limiting disease.

The proposal from the National Institute for Health and Care Excellence is only in draft form.

NICE said it was satisfied that the treatment would enable the patients to achieve their normal developmental milestones – and it was possible it would bring them close to a total cure.

The treatment, onasemnogene abeparvovec, will be given to babies diagnosed in infancy with type 1 SMA and also to babies diagnosed with three copies of the SMN2 gene.

Other children who fall outside the guidelines will have their cases considered individually by a national multidisciplinary clinical team.

The treatment is the second gene therapy adopted in the NHS using the adeno-associated virus vector.

Meindert Boysen, deputy chief executive of NICE, said: “Spinal Muscular Atrophy is a very serious, debilitating and distressing condition that has very significant effects on every aspect of life of those with SMA, and their families and carers. Being able to support access to one of the most exciting recent innovations in life sciences, a one-off therapy designed to address the genetic root cause of SMA, is both humbling and rewarding.

“The committee concluded that onasemnogene abeparvovec represents an important development in treating SMA which could not only allow babies to gain important motor milestones such as independent sitting and walking, but for some babies who are diagnosed before they have symptoms, it might come close to being a cure.”

Tags: Brain & Neurology | Child Health | Genetics | NHS | UK News

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