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NICE backs first Duchenne muscular dystrophy drug

Reverses original decision to reject the therapy in favour of more data

NICE has reversed an earlier decision to reject PTC Therapeutics' Duchenne muscular dystrophy (DMD) drug Translarna, but has warned access to the pricey drug will have to be carefully managed.

In draft guidance issued on Friday, the UK's cost-effectiveness watchdog said it supported the use of exon-skipping therapy Translarna (ataluren) for ambulatory patients aged five and older with DMD caused by a 'nonsense' mutation.

NICE initially rejected Translarna in an earlier draft of its guidance last October, asking for more evidence for the size of the drug's benefit in DMD, a progressive muscle wasting disease that is usually fatal by age 30.

Children with the disease typically become wheelchair dependent by age 12 and NICE said ataluren has "the potential to delay the loss of ability to walk, one of the most important factors for patients and their families".

The agency described the review as "a difficult evaluation given the uncertain nature of the data and the high cost of the drug".

Given Translarna's list price of around £220,000 a year, "it is important that details of the financial and other arrangements to enable this new medicine to be offered to patients on the NHS are discussed and agreed between the company and NHS England, and set out in a managed access agreement," said chief executive Sir Andrew Dillon.

PTC has offered an undisclosed discount on the list price to NHS England and Translarna will be offered under an initial five-year agreement in which the company will be expected to generate additional data on its efficacy.

At the moment, boys with muscular dystrophy are typically treated with corticosteroids, which can alleviate some symptoms but also have serious side effects such as growth retardation, bone thinning, mood swings and weight gain.

Exon-skipping therapies such as Translarna are the first to strike at the underlying causes of the disease. PTC Therapeutics' drug was the first drug in this class to be approved by the European Commission in 2014, although it was rejected by the FDA earlier this year with a request for more data.

It is thought around 50 children in the UK could benefit from the drug during the five-year managed access agreement, according to NICE.

The charity Muscular Dystrophy UK, which has been fighting for Translarna to be made available on the NHS, said the announcement was "wonderful news and a true victory for the families", although it expressed concern that there may now be a delay before the drug is available in clinics.

"We call on NHS England to act with the urgency and resolve that these children and their families deserve," said the charity's CEO Robert Meadowcroft.

Article by
Phil Taylor

18th April 2016

From: Regulatory



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