The NHS should pay for Novartis’ £1.79 million one-off gene therapy for Spinal Muscular Atrophy (SMA) NICE has said, the most expensive treatment ever approved for funding.
In first draft guidance NICE said Zolgensma (onasemnogene abeparvovec) should be made available for babies up to 12 months with type 1 SMA, although the age restriction is not present in the drug’s EU marketing authorisation.
This is one of the severest forms of the inherited condition and the life-expectancy of people 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The draft guidance also recommends treatment of presymptomatic babies with SMA who have up to three copies of the SMN2 gene as part of a managed access arrangement while further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 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.
NICE and Novartis have agreed a simple discount, which is commercially confidential, to the list price.
This is the second gene therapy recommended for funding by NICE – in 2019 Novartis’ Luxturna (voretigene neparvovec) was approved for NHS funding for a rare inherited eye disease.
Because of the limited trial data for children aged seven to 12 months, NICE said their treatment should be discussed by a national multidisciplinary clinical team.
NICE considered the treatment under its Highly Specialised Technologies programme, which uses a higher threshold for cost-effectiveness, reflecting the difficulties for developing treatments for extremely rare conditions that might only affect a handful of patients annually.
Children with type 1 SMA who fall outside these recommendations, but within the scope of the drugs marketing authorisation, may also be considered for treatment by a national multidisciplinary team.
Where the team says treatment with Zolgensma would be clinically appropriate, NHS England will reimburse the costs.
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.
SMA affects the nerves in the spinal cord controlling movement. This causes muscle weakness, progressive loss of movement, and difficulty breathing and swallowing.
Zolgensma uses a modified adeno-associated virus vector to carry a functional copy of the SMN1 into motor neurones, to promote their survival and function.
NICE already recommends Biogen’s Spinraza (nusinersen) CHECK for some people with pre-symptomatic SMA and types 1, 2 or 3 SMA as part of a managed access agreement.
For people who can’t have Spinraza, their condition is managed through supportive care which aims to minimise the impact of disability, address complications and improve quality of life.
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