UK regulators have renewed the scientific opinion for Santhera’s Raxone, extending its availability to patients with Duchenne muscular dystrophy (DMD) through the Early Access to Medicines Scheme (EAMS) for another year.
The aim of EAMS is to provide patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need.
Inclusion in EAMS allows eligible patients with DMD in respiratory function decline who are not taking glucocorticoids to access to Raxone (idebenone) while Santhera is preparing to re-file its marketing authorisation in Europe.
Raxone has been available in the UK through EAMS since June 2017, and currently over 40 DMD patients are enrolled in the scheme at several specialised DMD centers in the UK.
However, in January this year the European Medicines Agency’s Committee for Medicinal products for Human Use (CHMP) concluded that an approval for Raxone in DMD could not be granted on evidence included in the submission.
It acknowledged the positive outcome of the Phase III DELOS trial - which the firm said offers statistically significant and clinically relevant evidence that Raxone (idebenone) slows the decline of respiratory function in DMD patients - but said it needed more data to further link the observed treatment effects on respiratory function outcomes to patient benefit.
“We are very pleased about the renewal of the EAMS for Raxone which allows patients with DMD who have progressed to the stage of respiratory decline and have no alternative therapeutic options available, to receive treatment,” said Thomas Meier, Santhera’s chief executive.
“The MHRA renewal comes as a sign of continued recognition of both the high unmet medical need of DMD patients and the positive benefit-risk of Raxone in this population."