Santhera Pharmaceuticals’s Raxone has been renewed in UK, as the nation’s Medicines and Healthcare products Regulatory Agency (MHRA) has renewed the Early Access to Medicines Scheme (EAMS) scientific opinion for Santhera’s idebenone for patients with Duchenne muscular dystrophy (DMD) in respiratory function decline who are not taking glucocorticoids.
Santhera said that, at the occasion of the annual review, the MHRA confirmed its positive scientific opinion for Raxone under the EAMS and renewed the EAMS for a further 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 authorization when there is a clear unmet medical need. Inclusion in EAMS allows eligible patients with DMD, who meet criteria defined under this scheme, to gain access to Raxone, an investigational medicinal product for which Santhera is currently preparing marketing authorization applications in Europe and the USA, the company said.
Furthermore, Raxone has been available in the UK through EAMS since June 2017. At the present time over 40 DMD patients are enrolled in EAMS at several specialized DMD centers in the UK.
“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 CEO. He said that 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 that population. He said that the company is 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.
Santhera has quoted Janet Bloor, the chair of Action Duchenne and the mother of a young man living with Duchenne, as saying: “We have waited many years to realize the opportunity for early access to potential new treatments. It has taken a company like Santhera to focus on a huge unmet need and seek to address the underlying problems with respiratory failure. Time is something we don’t have with Duchenne and I welcome the renewal of the EAMS by the MHRA after effective consultation with the patient community.”