PTC Therapeutics’ DMD Drug Translarna Discounted for UK Access

The UK’s drug pricing watchdog is recommending reimbursement of PTC Therapeutics’ Translarna to treat Duchenne muscular dystrophy, provided the company complies with an access agreement.

Under the deal, PTC Therapeutics will offer the drug at a discounted price to patients aged 5 and older who can walk, if they participate in clinical assessments twice within a 14-month period to evaluate the drug. The amount of the discount was not disclosed.

The agreement also waives a three-month funding period, which could make the product available to patients within weeks, the UK’s National Institute for Health and Care Excellence said. The product’s availability gives patients with limited options a new potential treatment.