NICE requests more evidence on Duchenne muscular dystrophy therapy
pharmafile | October 16, 2015 | News story | Medical Communications, Sales and Marketing | Duchenne Muscular Dystrophy, NICE, PCT Therapeutics, Translarna, ataluren
NICE has chosen not to recommend has PTC Therapeutics’ treatment Translarna for Duchenne muscular dystrophy (DMD), asking for further clarification from the company on its efficacy.
The Institute has asked New Jersey-based PTC to provide the results of a confirmatory study of Translarna (ataluren) as soon as possible, and to provide further justification for the cost of the drug.
However the drug’s prospects have been hit by a further blow, after PTC announced that the drug failed to help boys with the muscle-wasting disease walk better in a final-stage clinical trial.
In a trial of 228 patients, there was no statistically significant difference in the distance boys could walk in six minutes between those given Translarna and those who had a placebo treatment.
Patients with moderate disease were most likely to benefit, going 47 metres farther during the walk test, which was a significant difference. None of the boys in this subgroup lost their ability to walk during the trial, compared with four getting a placebo. PTC also pooled together data from the Phase III study and a previous study, and found the difference in the six-minute walk test was a significant benefit of 22 metres.
DMD is one of a group of muscular dystrophies: inherited genetic conditions that cause the body to produce too little dystrophin, a substance crucial for muscle functioning. A lack of dystrophin causes the muscle fibres to weaken, which gradually weakens the muscles, resulting in an increasing level of disability. The decline in physical functioning in DMD leads to respiratory and cardiac failure and eventual death, usually before the age of 30.
DMD was the subject of an MP’s question to UK Prime Minister David Cameron on Wednesday, who was asked to guarantee that NICE would approve the drug on the NHS and offer the same level of patient access to Translarna as in Scotland, where the drug is available.
NICE’s draft guidance relates to people with DMD with a mutation (known as a nonsense mutation) in the dystrophin gene. Between eight and 13 boys are born with the rare condition each year in the UK, which rarely affects girls.
Translarna is the first licensed treatment for DMD that addresses the loss of dystrophin, the underlying cause of the condition. It has a conditional marketing authorisation in the UK for the treatment of DMD resulting from a nonsense mutation in the dystrophin gene, in patients who are able to walk aged five years and older.
Commenting on the draft guidance, Professor Carole Longson, NICE Health Technology Evaluation Centre director, says NICE had not been convinced that “the proposed cost of ataluren was justified by the evidence”, and had been looking for a treatment that had prolonged the time before people with the condition need a wheelchair.
“We are disappointed not to be able to recommend ataluren in this draft guidance. DMD is one of the most common and severe forms of muscular dystrophy. The Committee heard from the patient experts that one of the most important aspects of managing DMD is maintaining their child’s ability to walk because this means they can continue to go to school independently and participate more fully in social and sporting activities with family and friends. There is, therefore, a clear need for a treatment that can prolong a child’s ability to walk but without serious side-effects.
“After considering the evidence, and the opinions of the clinical and patient experts, the Committee agreed that ataluren represents an important development in the treatment of DMD and could potentially prolong the time before children have to use a wheelchair, compared with best supportive care.
“However, the Committee was not convinced that the proposed cost of ataluren was justified by the evidence presented on the additional health benefits associated with ataluren over standard therapy. Therefore, on the basis of the current evidence, the Committee was minded not to recommend ataluren for treating nonsense mutation DMD.”
The results of a confirmatory study of ataluren are due to be available shortly, and this will be presented to NICE along with further clarification and “justification of the cost of the drug”.
Joel Levy
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