Translarna not approved by NICE for DMD on NHS

In draft guidance published on Friday, it emerged NICE does not recommend PTC Therapeutics’ Translarna (ataluren) for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation.

The decision came following a re-evaluation of Translarna in order to decide whether to make it available for routine funding on the NHS. The re-evaluation followed a six-year period during which the DMD drug had been available under a managed access agreement (MAA), which ends in January next year, and under which 60 children have had access to Translarna.

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. One of a group of muscular dystrophies, DMD leads to a gradual loss of muscle function, often with children having to use a wheelchair by their early adolescence. Eventually, they will need assistance with breathing.

Translarna was the first licensed treatment for DMD which addresses the loss of dystrophin. A gene therapy, it permits the body to read over the DNA mutation and still continue to produce dystrophin.

After review of all data, including RWE, feedback from patients and clinicians and caregivers, as well as that collected from the MAA – NICE released its draft guidance based on “limited and highly uncertain” evidence of improved later stages of the disease or improved survival with Translarna.

Nonetheless, the committee agreed that the ALS drug can improve outcomes, including the prevention of scoliosis and maintaining upper body strength after patients have lost the ability to walk.

Interim director for medicines evaluation at NICE, Helen Knight said: “After considering all the available evidence, and the opinions of the clinical and patient experts, the committee concluded that the benefits provided by ataluren [Translarna] over standard treatment do not justify the current cost.”

Priced at £220,000 per year, NHS England and PTC Therapeutics have arranged for those currently receiving Translarna under the MAA to continue to be able to have it as long as it is of clear benefit to them.

Translarna has had European approval for usage since 2014 and has also been available under terms similar to the MAA to DMD patients in Scotland since 2021.

Last week, Sarepta Therapeutics filed for accelerated FDA approval of its DMD gene therapy developed with Roche, SRP-9001 (delandistrogene moxeparvovec).