DMD patients in the UK win early access to Santhera’s Raxone

Santhera Pharmaceuticals’ Raxone has become the first drug for Duchenne Muscular Dystrophy (DMD) to join the UK’s Early Access to Medicines Scheme (EAMS).

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted Raxone (idebenone) a positive scientific opinion for the treatment of DMD patients with respiratory function decline not taking glucocorticoids.

The decision allows patients who meet criteria to gain access to the drug before European regulators rule on whether it should be allowed onto the market. 

"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, chief executive 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."

An application to market Raxone for DMD was filed with the European Medicines Agency in June last year, based on data from the Phase II (DELPHI) programme and successful pivotal Phase III (DELOS) study, which showed a statistically significant and clinically relevant benefit of the treatment in preserving respiratory function compared to placebo.

The findings were also substantiated by a natural history study showing that the benefit observed in the Raxone-treated group would not have been expected from the natural course of the disease, the firm said at the time.

Also, it noted the marketing authorisation includes data showing that patients taking the drug in the DELOS trial had a reduced risk of bronchopulmonary complications including fewer hospitalisations caused by such complications and a reduced need for systemic antibiotic treatment compared to patients receiving placebo.

Raxone won European approval last year as the first treatment for visual impairment in adolescent and adult patients with Leber's Hereditary Optic Neuropathy (LHON), an inherited mitochondrial disease which, if untreated, usually leads to rapid, profound and permanent blindness.