The U.K.’s National Institute for Health Care and Excellence (NICE) has recommended for National Health Service (NHS) use PTC Therapeutics’ Translarna (ataluren) to treat ambulatory individuals two years and older with Duchenne muscular dystrophy (DMD) resulting from a nonsense mutation in the dystrophin gene.

NICE’s recommendation for Translarna was supported by evidence suggesting that the drug likely slows down disease progression and delays the loss of the ability to walk. But the agency  cited uncertainty for later stages of disease and for improved survival.

Ataluren has conditional marketing authorization in the UK that requires the company to provide confirmatory trial data on its effectiveness and safety.

The UK list price of Translarna is approximately $245,000 per patient annually. The negotiated price of the drug for NHS use was not disclosed.