NICE Approves Life-Changing Gene Therapy for Spinal Muscular Atrophy

Priscilla Lynch 

March 10, 2021

A new and potentially curative one-off gene therapy for babies with the rare genetic disorder spinal muscular atrophy (SMA) is set to become the most expensive treatment ever approved by the National Institute for Health and Care Excellence (NICE).

NICE’s newly published draft guidance recommends the £1.79 million treatment onasemnogene abeparvovec (Zolgensma, Novartis) for babies aged up to 12 months with type 1 SMA. This is one of the severest forms of the inherited condition, and the life expectancy of people with it is usually less than 2 years.

The draft guidance also recommends treatment of pre-symptomatic babies with SMA who have up to three copies of the SMN2 gene as part of a managed access agreement while further data are collected.

Despite the high cost of the treatment, NICE said it can be recommended for use on the NHS because of the evidence of exceptional benefit to young babies, potentially allowing them to reach normal childhood developmental milestones. Because of the limited trial data for children aged seven to 12 months, their treatment should be discussed by a national multidisciplinary clinical team.

Onasemnogene abeparvovec will become the second gene therapy recommended by NICE which uses the adeno-associated virus (AAV) vector. In this case, it is modified to carry a functional copy of the SMN1 gene into the motor neurons with the aim of promoting their survival and function. It is given as a one-off infusion and its effects are thought to be lifelong.

The treatment was considered under the NICE Highly Specialised Technologies programme which uses a higher threshold for cost-effectiveness to reflect the difficulties of developing treatments for extremely rare conditions that might only affect a handful of patients per year.

Around 65 babies are born each year in England with SMA, of whom about 60% are diagnosed with type 1 SMA.

The draft guidance is open for public consultation until 6 April.

This article originally appeared on Univadis, part of the Medscape Professional Network.

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