MODERATE IMPACT – 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.

  • Translarna is licensed in multiple countries, including Great Britain, Northern Ireland, and the European Economic Area, for treating nonsense mutation DMD in ambulatory patients aged 2 years and older.
    • Ataluren remains an investigational new drug in the United States.
  • The final evaluation document stated that the cost-effectiveness estimates were uncertain because of the limitations in the clinical effectiveness data. But, with the commercial arrangement agreed after the second committee meeting, the cost-effectiveness estimates for ataluren are below the range that NICE usually considers acceptable for highly specialized technologies. Therefore, ataluren is recommended.

inThought Analysis

In October 2022, NICE’s draft guidance was published not recommending Translarna to be covered for DMD, stating that benefits did not justify the cost. The final evaluation document states that Translarna was only recommended because of a commercial arrangement that makes Translarna available at a discount. The details of the discount are confidential.

At JPM 2023, PTC announced it achieved $289M in revenue for Translarna, with growth driven by new patients in existing geographies and continued geographic expansion. The NICE evaluation and agreement with NHS will help solidify newly diagnosed and existing patient access to Translarna in England and Wales, but how the magnitude of the discount will affect revenue is unclear. PTC continues to seek approval in the U.S., but the FDA’s concerns over efficacy remain.


Source: PTC Press Release, NICE Final Evaluation Report