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ataluren (Translarna®)

Reference No. 940

Publication date:

Appraisal information

ataluren (Translarna®) 1000 mg granules for oral suspension
ataluren (Translarna®) 125 mg granules for oral suspension
ataluren (Translarna®) 250 mg granules for oral suspension

Company: PTC Therapeutics Ltd
BNF category: Musculoskeletal and joint diseases
NMG meeting date: Not scheduled
AWMSG meeting date: 13/07/2016
Submission Type: NICE HST
Status: Recommended
Advice No: Not available
Ratification by Welsh Government: 19/08/2016

Current Progress

Ratification by
Welsh Government

AWMSG advice

The National Institute for Health and Care Excellence (NICE) published advice in July 2016 in relation to ataluren (Translarna®) for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older. At their meeting on 13th July 2016 AWMSG supported the adoption and implementation of this advice within NHS Wales (refer to the minutes of meetings). Welsh Government has subsequently agreed that the NICE HST advice for ataluren (Translarna®) should be implemented within NHS Wales. Refer to the NICE website for full guidance including any specific restrictions on the use of the technology.