MHRA keeps Santhera's Raxone available to patients with 'clear unmet need'

Santhera Pharmaceuticals announced on Friday that the UK's Medicines and Healthcare products Regulatory Agency (MHRA) has renewed the Early Access to Medicines Scheme (EAMS) scientific opinion for ‘Raxone’ (idebenone), for patients with Duchenne muscular dystrophy (DMD) in respiratory function decline who are not taking glucocorticoids.

The Swiss drugmaker said at its annual review, the MHRA confirmed its positive scientific opinion for Raxone under the EAMS, and renewed the EAMS for a further year.

It said the aim of EAMS was to provide patients with life threatening or seriously debilitating conditions access to medicines that did not yet have a marketing authorisation, when there was a “clear unmet medical need”.

Inclusion in EAMS allowed eligible patients with DMD, who met criteria defined under the scheme, to gain access to Raxone, which Santhera said is an investigational medicinal product for which it was currently preparing marketing authorisation applications in Europe and the US.

Raxone has been available in the UK through EAMS since June last year.

Currently, more than 40 DMD patients were enrolled in EAMS at several specialised DMD centers in the UK, the company reported.

"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 Santhera CEO Thomas Meier.

“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.

“We are committed to addressing this urgent medical need and will make Raxone available to all patients in the UK who meet the eligibility criteria for the EAMS upon request of their treating physician.”