NHS Refuses Translarna For The Treatment of Duchenne Muscular Dystrophy

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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A scientist who helped develop a pioneering treatment recently expressed her frustration after the NHS refused funding of Translarna for the treatment of Duchenne Muscular Dystrophy.

Translarna has recently become the first Duchenne Muscular Dystrophy targeting therapy to receive approval by the European Medicines Agency (EMA). The drug is available in many countries within the European Union, however is still to be available in England.

Since last summer, many families of patients with Duchenne Muscular Dystrophy are waiting for NHS approval to potentilly fund the drug which could help children with the condition walk longer and slow muscle wasting progression.

NHS England has recently announced the deferral of its response to the National Institute for Health and Care Excellence (NICE). NICE will conduct a drug review, which should be reported back in early 2016.

Professor Kate Bushby from the The John Walton Muscular Dystrophy Research Centre (part of the Institute of Genetic Medicine at Newcastle University) has been leading a clinical trial of Translarna. Professor Bushby, Honorary Consultant Geneticist at Newcastle Hospitals NHS Foundation Trust, stated in a recent news release: “It is very disappointing for the Duchenne muscular dystrophy community that the NHS has decided not to fund Translarna at this juncture. The drug is already available in several European countries following EMA conditional approval last year including Germany, Greece, Italy and France. Drugs for rare diseases are very expensive, but this is a function of the development pipeline and should not disadvantage the patients who suffer from these conditions. If we are to have a constructive pipeline for rare disease drug development then there needs to be a way to ensure that drugs which have been approved by the EMA have a mechanism to be available on the NHS”.

“We in the UK led Europe in recruitment for the clinical trials of Translarna and it is very disheartening to see that the investment that the research community, and the time given up by patients and families to participate in these studies is not being reflected in a positive funding decision. There are likely to be many drugs coming on line for Duchenne where the benefit will be to stabilise the disease or reduce the rate of progression and these outcomes in themselves are extremely valuable in such a relentlessly progressive condition”, she added.

Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness and is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptom onset occurs during early childhood, usually between ages 3 and 5. The disease primarily affects boys, but in rare cases it can affect girls.

The drug is only suitable for the treatment of 10 to 15% of those patients suffering with the diease and its mechanism of action allow the body to compensate for the genetic mutations that cause the disease.

In October, NICE will produce a draft guidance on the treatment, with the final guidance expected shortly after. Importantly, further studies on the drug efficacy are expected to be complete in October.

Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK, said in the news release: “This news is a harsh blow to each and every boy and young man living with Duchenne muscular dystrophy. Translarna is the first drug licensed and available in the EU to treat the underlying genetic cause for a proportion of boys with this devastating condition. Parents of young children have faced a grueling nine-month wait for an answer on funding, only to hear ‘no’ from NHS England. Translarna could give boys precious extra time walking for longer, helping to delay the progression of the condition. Some boys are now at high risk of losing the ability to walk before NICE’s assessment next year and ultimately, once this has happened these boys will also lose the opportunity to take this drug. Our focus now is to support parents affected by this decision through the waiting period, and to explore all alternatives, including working with senior doctors on requests for individual funding. We will also be looking ahead to NICE’s decision in early 2016, building the case for funding for Translarna.”