A gene therapy for treating children with Duchenne muscular dystrophy caused by a ‘nonsense’ mutation has been provisionally rejected by NICE.
In its draft guidance, published today, it says following a re-evaluation, it does not recommend ataluren, which is also called Translarna.
This follows a six-year period during which it was available under a managed access agreement (MAA). This agreement, under which about 60 children have been prescribed ataluren, ends in January 2023.
Ataluren, manufactured by PTC Therapeutics, is the first licensed treatment for DMD that addresses the loss of dystrophin by allowing the body to read over the mutation in the DNA and continue to produce dystrophin.
The data collected as part of the MAA, plus new real-world evidence on ataluren and feedback from clinicians and patients, was reviewed for the draft guidance.
The data suggest while ataluren is likely to slow down disease progression and delay the time at which the ability to walk is lost, evidence for improvements in later stages of the disease and improved survival with ataluren is limited and highly uncertain.
However, the committee says ataluren, which costs about £220,000 per patient per year at its list price, may also improve outcomes, such as preventing scoliosis and maintaining upper body strength, once the ability to walk has been lost.
The committee disagreed with some of the company’s approaches to the economic modelling for ataluren. Members said it could not account for a benefit of treating children from the age of two years when it is rare to diagnose the condition at that age.
It also considered the company’s modelling assumptions for treatment stopping rates and stopping rules were unrealistic and underestimating the costs of ataluren.
Members said after taking into account all of the evidence, the cost-effectiveness estimates for ataluren are substantially above the range NICE considers acceptable for highly specialised technologies.
Helen Knight, interim director for medicines evaluation at NICE, said: “We know that today’s provisional decision not to recommend ataluren will be disappointing for people with this devastating condition and to the people who care for them.
“However, after considering all the available evidence, and the opinions of the clinical and patient experts, the committee concluded that the benefits provided by ataluren over standard treatment do not justify the current cost of ataluren.”
NHS England and PTC Therapeutics have agreed that individuals currently having ataluren as part of the MAA can continue to be able to access the treatment for as long as clinicians view them to be beneficial, even if NICE is unable to make a positive final recommendation.
Newly diagnosed patients will also be able to start and continue treatment up to the end of the MAA.
The closing date for comments is 21 October 2022 and the second evaluation committee meeting will take place on 15 December 2022.
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