Duchenne drug available in England within weeks after NICE approval

PTC Therapeutics’ Translarna (ataluren) will be available to Duchenne muscular dystrophy patients “within weeks”, NICE has said, after a deal was agreed with the manufacturer.

NHS England struck a “managed access agreement” with PTC and has waived a three-month waiting period for funding.

The decision is a hugely significant step forward for sufferers, as Translarna is the first ever drug for the condition to gain NICE approval.

The ruling puts England at the forefront of treating the disease - the FDA rejected a filing for Translarna earlier this year and has concerns about the efficacy of rival treatments, meaning no drug for Duchenne is available in the US.

The cost-effectiveness body in final guidance recommended Translarna, which has a list price of £220,000 per year, for children aged five and over with DMD caused by a nonsense mutation.

NHS England and PTC finalised the five-year deal earlier this month, which include an outcomes-based incentive for the manufacturer and a mechanism to monitor how well the medicine has worked in practice before future funding decisions are taken.

The agreement also included a confidential discount, NICE said.

Translarna has been called a “step change” in the management of the rare muscle wasting disease, which is usually fatal by age 30.

Duchenne muscular dystrophy is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. Usually affecting only boys, there are between 60 and 70 children born with the disease in England each year and in around 6 – 9 children (13%) it is caused by a ‘nonsense’ mutation

Children with DMD are typically dependent on a wheelchair by age 12, and NICE’s appraisal committee agreed the drug could extend this by up to seven years.

This could give children a normal adolescence and allow them to continue school for longer, NICE said.

Standard treatment is corticosteroids which can delay deterioration but can cause unwanted effects such as growth retardation, bone thinning, mood swings and weight gain.

A so-called exon-skipping drug, Translarna works by allowing the body to read over the mutation in the DNA and continue to produce dystrophin.

Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said: “Families in England will deservedly be thrilled by today’s news from NICE. Together, we have fought long and hard for this pioneering drug to be made available on the NHS. For all those who campaigned with such determination, today is a very welcome relief.”

In the US, regulators have asked for more data on Sarepta’s DMD drug, eteplirsen in a long-running saga where the FDA came under pressure from patients and politicians to approve the drug after advisers said to reject it.

Santhera will not get a decision on its DMD drug, Raxone (idebenone) until 2020 and BioMarin in May axed development of its Kyndrisa (drisapersen) and three follow-on drugs, after rejection by regulators in the US and EU.