24 June 2019 - Santhera Pharmaceuticals announces that the UK's Medicines and Healthcare products Regulatory Agency (MHRA) has renewed for a further year the Early Access to Medicines Scheme scientific opinion for idebenone for patients with Duchenne muscular dystrophy in respiratory function decline who are not taking glucocorticoids. 

With this renewal, the MHRA again confirmed its positive scientific opinion for idebenone under the EAMS while a corresponding European marketing authorisation application has recently been submitted.

When applying for the EAMS renewal, Santhera submitted new efficacy data (including results from the long-term SYROS study) supporting the potential for a clinically relevant preservation of respiratory function during idebenone treatment for up to six years in a real-world setting. By renewing the EAMS, the MHRA has enabled access to idebenone for Duchenne muscular dystrophy patients with the highest need. Recently, Santhera has submitted a conditional marketing authorisation application to the EMA for idebenone (under the trademark Puldysa) to treat respiratory function decline in DMD.

Read Santhera Pharmaceuticals press release