MHRA in the UK grants Raxone (idebenone) a positive scientific opinion to treat Duchenne Muscular Dystrophy under Early Access to Medicines Scheme.- Santhera Pharmaceuticals

Santhera Pharmaceuticals announces that the UK's Medicines and Healthcare products Regulatory Agency (MHRA) has granted Raxone (idebenone) a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) for patients with respiratory function decline not taking glucocorticoids in Duchenne Muscular Dystrophy (DMD).

The aim of the 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. The MHRA decision allows patients with DMD, who meet criteria defined under this scheme, to gain access to Raxone, an investigational medicinal product currently under review for DMD for Marketing Authorization by the European Medicines Agency (EMA). Under the EAMS, and as shown in the public assessment report, Raxone is indicated for slowing the decline of respiratory function in patients with DMD from the age of 10 years who are currently not taking glucocorticoids. The decline of respiratory function must be confirmed by repeated measurements prior to initiation of treatment. Raxone can be used in patients previously treated with glucocorticoids or in patients in whom glucocorticoid treatment is not tolerated or is considered inadvisable.

The UK's industry-sponsored EAMS aims to give 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. Under the scheme, the MHRA provides a scientific opinion on the benefit/risk balance of the medicine, based on the data available when the EAMS submission was made. The opinion lasts for a year and can be renewed. The scheme is voluntary and the opinion from MHRA does not replace the normal licensing procedures for medicines.