First DMD Patients to Receive Raxone Under U.K.’s Early Access Program

Magdalena Kegel avatar

by Magdalena Kegel |


The first patients with Duchenne muscular dystrophy (DMD) have been enrolled into the U.K.’s Early Access to Medicines Scheme (EAMS) for Santhera Pharmaceuticals’ Raxone (idebenone), giving patients access to the treatment before its regulatory approval.

Raxone was included in EAMS after the Medicines and Healthcare Products Regulatory Agency (MHRA) granted Raxone a positive scientific opinion in June.

“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,” Dipansu Ghosh, MD, a respiratory physician at a Duchenne center in Leeds, said in a press release.

The decision to grant patients early access to Raxone through the EAMS also was applauded by Action Duchenne, an organization that supports research into treatment and cure of Duchenne.

“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,” said Janet Bloor, Chair of Trustees at the organization.

“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,” she added.

So far, 15 specialist Duchenne centers in the U.K. have obtained the training required by the EAMS to allow Raxone treatment. Additional centers are in line to receive training.

Raxone — a drug that aims to treat respiratory function decline in Duchenne patients by improving energy production in cellular power plants called mitochondria — is under review by European regulators.

In June 2016, the European Committee for Medicinal Products for Human Use (CHMP) validated a marketing application for Raxone as a treatment for Duchenne muscular dystrophy patients with respiratory function decline who are not treated with glucocorticoid therapy.

In May 2017, Santhera, however, changed the application to include patients who have been treated previously with glucocorticoids, and patients for whom glucocorticoid treatment is not a viable option.

These aspects are included in the EAMS approval, which approves of treatment for children 10 years or older with a confirmed decline of respiratory function.

While Santhera has backed its marketing application with data from two clinical trials, the Phase 2 DELPHI study (NCT00654784) and the Phase 3 DELOS (NCT01027884), the company launched another Phase 3 study in February 2017.

This study, called SIDEROS (NCT02814019) will further assess Raxone’s effectiveness in slowing respiratory decline regardless of glucocorticoid use.