The Medicines and Healthcare Products Regulatory Agency (MHRA) has renewed its Early Access to Medicines Scheme (EAMS) scientific opinion for a further year, for Santhera’s Puldysa (idebenone) in Duchenne muscular dystrophy.
The indication is 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.
The drug can also be offered to patients previously treated with glucocorticoids or in patients in whom glucocorticoid treatment is not tolerated or is considered inadvisable.
The renewal means that the MHRA has again confirmed its positive scientific opinion for the drug under the EAMS while a corresponding European marketing authorisation application (MAA) has recently been submitted.
Santhera welcomes the EAMS renewal “as it provides a therapeutic option for DMD-patients with deteriorating respiratory function in the UK who really have no treatment alternative,” said Kristina Sjöblom Nygren, chief medical officer and head of development at Santhera.
She continued, “Last month, we submitted a European MAA for idebenone in DMD. In this context, the MHRA renewal not only comes as a continued recognition of the positive benefit-risk of idebenone in this patient population but also helps bridge the time until drug approval.”
The drug has been available in the UK through EAMS since June 2017, where at present 67 patients with DMD are benefiting from access to idebenone through EAMS at several specialised DMD centres.