The UK’s National Institute for Health and Care Excellence has recommended reimbursement for PTC Therapeutics’ Translarna for the treatment of Duchenne muscular dystrophy.
According to draft guidance, the cost watchdog determined the drug is an acceptable value for NHS England — provided that PTC delivers a promised pricing discount — because of its potential benefits and the lack of a proven alternative. Translarna is estimated to cost approximately $314,300 per patient per year.
PTC’s Managed Access Agreement with NICE is expected to allow the company a five-year period to collect further data on Translarna’s efficacy. The guidance will be reviewed again at the end of that period, the drugmaker says.