Decision signed after revocation of the marketing authorisation by the EU Commission

To ensure continuity of treatment for those already undergoing treatment with Translarna for Duchenne muscular dystrophy, following the European Commission's failure to renew the medicine's conditional marketing authorisation, it is possible, at the request of the treating physician, to start the procedure for importing Translarna from abroad. This was decided by the Italian Medicines Agency, expressing closeness to patients and their families. The cost will be borne by the regional health services, without any costs for patients.

President Robert Nisticò has signed the Decision authorising the temporary supply of the medicine, no longer marketed, with the aim of ensuring therapeutic continuity for Italian patients. The treatment may be administered, “on the basis of a nominal medical prescription and under the responsibility of the same prescribing physician”, only to “ambulant patients, already undergoing treatment and for a maximum period of six months, in accordance with the provisions of European Directive 2001/83”, specifies the Decision, in force from the day after publication in the Official Journal.

The initiation of this procedure was made possible thanks to Aifa's efforts to have the European Commission offer member states the possibility of adopting, on an exceptional basis, measures to ensure the continuation of the therapy, after the decision to revoke the authorisation by transposing the Ema's indication based on some recent studies that did not confirm the efficacy of Translarna.