Eteplirsen for treating Duchenne muscular dystrophy [ID1003]
Discontinued
Reference number: GID-HST10007
As you will be aware, the Department for Health and Social Care has asked NICE to carry out an evaluation of eteplirsen for treating Duchenne muscular dystrophy.
However, the company have advised that during June 2018 the Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion, recommending the refusal of the marketing authorisation for eteplirsen for treating Duchenne muscular dystrophy. The company have already requested a data re-examination by the CHMP, the outcome of this will be known during the Autumn.
Therefore, as is usual practice with a negative CHMP opinion, NICE has decided to suspend this appraisal on its work programme but will continue to monitor the regulatory situation for eteplirsen and will update interested parties if the situation changes.