Santhera enrolls first n Duchenne Muscular Dystrophy patients in Raxone study

Santhera enrolls first n Duchenne Muscular Dystrophy patients in Raxone study

September 11, 2017 Off By Dino Mustafić

Santhera Pharmaceuticals has enrolled first DMD patients with respiratory function decline not taking glucocorticoids into UK’s Early Access to Medicines Scheme (EAMS) for Raxone, the company said Monday.

In June, Raxone (idebenone) was granted a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) – the first drug approved under the EAMS for patients with DMD.

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 Marketing Authorization in DMD by the European Medicines Agency (EMA).

Enrollment into the EAMS documents the first use of Raxone outside of a clinical trial for patients with DMD.

Dr. Dipansu Ghosh, a respiratory physician at a DMD center based in Leeds, said: “I am pleased to be able to offer Raxone to several of my patients in respiratory decline, as there are no other medical treatments available.”

Janet Bloor, Chair of Trustees of Action Duchenne, said: “At Action Duchenne we were encouraged by the positive EAMS opinion, earlier this summer. Particularly, for young people living with Duchenne who have respiratory decline. I am delighted that respiratory function is being considered by the regulatory agencies. This will hopefully pave the way for more potential treatments that may benefit the wider spectrum of DMD patients.”

Image: Bottles containing 180 tablets of Raxone (idebenone 150 mg). Image: Santhera