The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has renewed for a further year the Early Access to Medicines Scheme (EAMS) scientific opinion for Santhera’s Duchenne muscular dystrophy (DMD) drug idebenone.
The drug is being developed for DMD patients in respiratory function decline who are not taking glucocorticoids.
With this renewal, the MHRA has confirmed its positive scientific opinion for idebenone under the EAMS while a corresponding European marketing authorisation application (MAA) is currently under review.
The decision continues to facilitate access to idebenone for DMD patients with respiratory function decline, a leading cause of increased morbidity and early death for which no approved therapy exists.
A conditional marketing authorisation for the drug, under the tradename Puldysa, to treat is currently under regulatory review, with Santhera expecting an opinion by the Committee for Medicinal Products for Human Use (CHMP) in the fourth quarter of this year.
“This EAMS renewal for idebenone enables a much-needed therapeutic option for DMD patients with deteriorating respiratory function who have no real treatment alternative. We welcome the continued recognition by the UK’s MHRA of the positive benefit-risk of idebenone in this patient population,” said Kristina Sjöblom Nygren, Santhera’s chief medical officer and head of development.
Idebenone has been available in the UK through EAMS since June 2017. At present, 84 patients with DMD are benefiting from early access to the drugn through EAMS at several specialised DMD centres across the UK.
