NICE backs reimbursement for Duchenne drug Translarna

The UK health technology assessor, the National Institute for Health and Care Excellence (NICE) has issued a Final Evaluation Document recommending Translarna (ataluren) for reimbursement and use across the National Health Service (NHS) in England and Wales in the treatment of Duchenne muscular dystrophy (DMD).

Marketed by New Jersey, USA-based rare disease drug developer PTC Therapeutics (Nasdaq: PTCT), Translarna is the only approved treatment for patients with nonsense mutation Duchenne muscular dystrophy aged two years and older who can walk, the company noted.

Duchenne is a severe progressive disease that leads to rapidly worsening muscle function with children often using a wheelchair by early adolescence and eventually requiring artificial ventilation to breathe. The NICE recommendation was based on data from clinical trials and real-world evidence demonstrating Translarna's potential to slow disease progression and improve patient outcomes.