Tuesday, June 23, 2020 @ 7:00 am
Santhera Pharmaceuticals (SIX: SANN) announces that 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 idebenone for patients with Duchenne muscular dystrophy (DMD) 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 authorization application (MAA) is currently under review.
By renewing the Early Access to Medicines Scientific Opinion the UK’s Medicines and Healthcare products Regulatory Agency continues to enable access to idebenone for Duchenne Muscular Dystrophy patients with respiratory function decline, a leading cause of increased morbidity and early death for which no approved therapy exists. A conditional marketing authorization (CMA) application for idebenone (under the trademark Puldysa) to treat respiratory dysfunction in DMD is currently under regulatory review and Santhera expects an opinion by the Committee for Medicinal Products for Human Use (CHMP) in the fourth quarter 2020.
“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, MD, Chief Medical Officer and Head of Development at Santhera.
Idebenone has been available in the UK through EAMS since June 2017. At present, 84 patients with DMD are benefiting from early access to idebenone through EAMS at several specialized DMD centers across the UK.
Under the EAMS, as shown in the public assessment report, idebenone is indicated as a treatment for slowing the decline of respiratory function in patients with DMD from the age of 10 years who are currently not taking glucocorticoids. Patients will need to meet the clinical criteria for entry into EAMS, including showing evidence of active decline of respiratory function prior to initiation of treatment. Idebenone can be offered to patients previously treated with glucocorticoids or in patients in whom glucocorticoid treatment is not tolerated or is considered inadvisable.