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NICE recommends £1.79m gene therapy for spinal muscular atrophy in babies

Published on 08/03/21 at 11:10am

NICE is set to approve a new and potentially curative one-off gene therapy for babies with the rare genetic disorder spinal muscular atrophy (SMA), which will be the most expensive treatment ever okayed by the public body.

Draft guidance published by NICE today recommends the £1.79 million treatment, Novartis’ onasemnogene abeparvovec (Zolgensma), for babies aged up to 12 months with type 1 SMA.

SMA is a rare, progressive neuromuscular condition caused by a genetic mutation in the SMN1 gene on chromosome 5q. This causes a lack of survival motor neuron (SMN) protein, which causes motor neurones to malfunction, deteriorate and eventually die.

Type 1 SMA is one of the severest forms of the inherited condition, and the life-expectancy of those with it is usually less than two years.

The draft guidance also recommends treatment of presymptomatic babies with SMA who have up to three copies of the SMN2 gene, as part of a managed access arrangement while further data is collected.

Despite the treatment’s high cost, NICE have recommended the therapy for use on the NHS because of the evidence of exceptional benefit to young babies, potentially allowing them to reach normal childhood developmental milestones.

Onasemnogene abeparvovec will become the second gene therapy using the adeno-associated virus vector to be recommended by NICE. In this case, the treatment is modified to carry a functional copy of the SMN1 gene into the motor neurons with the aim of promoting their survival and function.

The therapy is given as a one-off infusion and its effects are thought to be life-long.

Meindert Boysen, Deputy Chief Executive and Director of the Centre for Health Technology Evaluation at NICE, said: “Spinal auscular 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.”

Darcy Jimenez

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