Raxone is first to receive positive opinion through the Early Access to Medicines Scheme

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

The EAMS was set up to offer possible therapeutic options to patients with life threatening or seriously debilitating diseases, where there is a clear unmet medical need, before they have achieved marketing authorisation.

This positive opinion will grant access to Raxone, which is an investigational drug currently under review by the European Medicines Agency (EMA), to DMD patients who meet criteria defined by EAMS. Raxone is indicated for patients (from the age of 10 years) with DMD who are currently not taking glucocorticoids and whose respiratory function is declining. It is specified that the decline of respiratory function must be confirmed by multiple measurements prior to treatment beginning. Patients who were previously on glucocorticoids of those in whom glucocorticoid treatment was not tolerated or considered inadvisable may also be suitable for treatment with Raxone under EAMS criteria.

“We’re proud to receive the positive EAMS scientific opinion for Raxone in the UK and to have our lead compound designated as a promising innovative medicine, the first for a drug intended for the treatment of DMD,” said Thomas Meier, PhD, CEO of Santhera. “This decision allows patients with DMD to receive treatment for respiratory function decline who otherwise would not have access to such treatment options.”

“This is excellent news for patients with respiratory decline in Duchenne muscular dystrophy,” added Janet Bloor, chair of the Board of Trustees at Action Duchenne. “The need for new treatments in DMD is very great and the EAMS can help to accelerate access for patients. Action Duchenne was pleased to provide advice during the development of the EAMS programme and we are delighted to see this first positive opinion in DMD.”

Santhera Pharmaceuticals is a Swiss speciality pharmaceutical company focused on the development and commercialisation of pharmaceutical products for the treatment of orphan mitochondrial and neuromuscular diseases.