An announcement was made by the Santhera Pharmaceuticals that the Medicines & Healthcare products Regulatory Agency of the United Kingdom has renewed the Early Access to Medicines Scheme for another year for idebenone for those patients who have Duchenne muscular dystrophy in breathing function deterioration who are in taking glucocorticoids.
Medicines & Healthcare products Regulatory Agency, with this renewal, has given confirmation of its affirmative scientific opinion for idebenone under the Early Access to Medicines Scheme while there has been a submission of an equivalent marketing authorization application of Europe.
While giving the application for the renewal of the Early Access to Medicines Scheme, Santhera made submission of fresh efficacy data inclusive of the results from the lasting SYROS study which further supported the prospective for clinically applicable protection of respiratory function during the treatment of idebenone for around 6 years in a setting of real-world. By the renewal of Early Access to Medicines Scheme, the Medicines & Healthcare products Regulatory Agency has allowed access to idebenone for the Duchenne muscular dystrophy (DMD) patients with the greatest need. Lately, a submission was made by Santhera of a conditional marketing authorization application to the European Agency of Medicines for idebenone for the treatment of the decline of respiratory function in the Duchenne muscular dystrophy.
The Chief Medical Officer, as well as, Head of Development, Santhera, Kristina Sjöblom Nygren, passed the following statement: “We hail the renewal of the Early Access to Medicines Scheme for idebenone as it gives provision of a therapeutic option for the patients with Duchenne muscular dystrophy with declining function of respiration in the United Kingdom who do not have any other alternative for treatment.” He continued: “A month ago, we submitted a European Market Authorization Application for idebenone in the DMD. In this situation, the renewal by MHRA helps to bridge the gap until the approval of the drug.”