Mother holding a young baby's hand

NICE approves spinal muscular atrophy gene therapy treatment

A new, potentially curative one-off gene therapy treatment for babies with spinal muscular atrophy (SMA), a rare genetic disorder, has been recommended for approval by NICE.

In their draft guidance, it recommends the £1.79m treatment for babies aged up to 12 months with type 1 SMA, which is one of the most severe forms of the inherited condition and leaves those affected with a life expectancy of typically less than two years.

The draft guidance also recommends the treatment for babies with SMA before they develop symptoms, as part of a managed access arrangement while further data is collected.

In approving the gene therapy, it is set to become the most expensive treatment ever approved by NICE.

Despite the high cost of the treatment, it is to be recommended for use on the NHS due to notable evidence of life-saving benefits it can provide to young babies, potentially allowing them to reach normal childhood developmental milestones.

Because of the limited trial data available for children aged 7-12 months, their treatment should be discussed by a national multidisciplinary clinical team.

Meindert Boysen, Deputy Chief Executive and Director of the Centre for Health Technology Evaluation at 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 [this treatment] 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.”

“As is the case with many new treatments for very rare diseases, limited evidence means there are uncertainties about the long-term benefits of [the gene therapy]. The collaborative effort of all involved in the evaluation has made it possible to recommend a treatment which, at its list price, is the most expensive drug NICE has ever evaluated, allowing the lives of babies who might otherwise have died before their second birthday to be transformed.”

In keeping with NICE standards, the draft guidance is open for public consultation until April 6, 2021.

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