The National Health Care Institute has advised the Minister of Health, Welfare and Sport (VWS) to end the conditional inclusion of ataluren (Translarna®) immediately. The effectiveness of ataluren has not been established. Ataluren has been conditionally included in the basic healthcare package since 1 November 2021. The medicine was used in the treatment of Duchenne muscular dystrophy. This is a rare and progressive muscular disease.
Ataluren is intended for certain people with Duchenne muscle disease
Ataluren is the active substance. The brand name is Translarna®. Ataluren is intended for the treatment of Duchenne muscular dystrophy due to a so-called nonsense mutation in the dystrophin gene in ambulatory patients aged 2 years and older. Duchenne muscular dystrophy is a rare and progressive muscular disease. To date, 15 Dutch patients have been granted access to ataluren through conditional inclusion.
Advice from the National Health Care Institute on early termination of conditional inclusion of ataluren
Ataluren is admitted to the basic healthcare package on a conditional basis from 1 November 2021 to 1 October 2025. The National Health Care Institute advises the Minister of VWS to terminate the CI procedure for ataluren immediately.
Ataluren was admitted to the market by the European Medicines Agency (EMA) subject to additional studies. After evaluating the additional study results, the EMA concludes that the effectiveness of ataluren has not been established. The registration of the medicinal product has, therefore, not been renewed. The medicine has been withdrawn from the European market and should no longer be supplied to patients. For all these reasons, ataluren can no longer be reimbursed through conditional inclusion. The National Health Care Institute has therefore advised the Minister to stop the CI procedure for ataluren immediately.