NICE Issues Guidance Recommending Translarna for Pediatric DMD

The National Institute for Health and Care Excellence (NICE) has just issued new guidance recommending Translarna (ataluren) for children older than 5 who have Duchenne muscular dystrophy (DMD).

The guidance, titled “Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene,” follows the announcement made on July 7, 2016, that the English National Health Service (NHS) and PTC Therapeutics had successfully negotiated a “managed access agreement” – a condition imposed by NICE to make the drug available – to set financial and clinical details regarding the use of Translarna.

Standard DMD treatments include corticosteroids, which can delay deterioration but also cause unwanted effects like growth retardation, bone thinning, mood swings, and weight gain. Translarna has been called a “step change” in this disease management since children with DMD typically become bound to a wheelchair by the age of 12, and the drug has the potential to extend this time by up to seven years.

Reports show that once a child loses his ability to walk, the deterioration worsens heavily and they eventually need help to feed themselves and for their personal care, like showering or using the toilet. If some time could be gained, experts believe that these children could have the opportunity to experience a more normal adolescence. This could also be a groundbreaking opportunity for these children to stay in school until later in life.

Translarna works by giving the body the ability to read over patient’s DNA mutation and continue to produce dystrophin. The drug’s list price is around £220,000 (U.S. $290,180) per year.

The committee took into consideration a clinical study in which no children in the most sensitive group lost the ability to walk over the 48 weeks of the trial, compared to 8 percent of the children in the placebo group. Patient experts reported meaningful stabilization of improvements in their child’s mobility, such as being able to get out of bed and go to school without any adult help.

“Duchenne muscular dystrophy caused by a nonsense mutation is a cruel disease that currently has few treatment options. Ataluren is an innovative drug that for the first time is aimed at the root cause of the disease and has the potential to offer benefits to people with the condition and their families,” NICE Chief Executive Sir Andrew Dillon said in a press release.

“When we published our draft recommendations in April we acknowledged that ataluren represents a significant cost to the NHS at a time of increased pressure on funding, especially given the uncertainties of the drug’s potential long-term benefits,” Dillon added.

He said NICE was “extremely pleased” NHS England and the company have agreed to the terms of a five-year managed access agreement for Translarna.

“The agreement means children with this condition will now be able to access the drug while at the same time allowing more data to be gathered on its efficacy, before the guidance is reviewed and a further decision made on whether funding should be continued after five years,” Dillon said.