The UK's Medicines and Healthcare products Regulatory Agency (MHRA) has granted Swiss company Santhera Pharmaceuticals’ a positive scientific opinion for its Raxone (idebenone) through the Early Access to Medicines Scheme (EAMS).

Raxone can be used to treat patients with respiratory function decline who are not taking glucocorticoids in duchenne muscular dystrophy (DMD).

The investigational medicinal product for DMD is currently under review for marketing authorisation by the European Medicines Agency (EMA).

Santhera Pharmaceuticals chief executive officer Dr Thomas Meier said: “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.

“This decision allows patients with DMD to receive treatment for respiratory function decline who otherwise would not have access to such treatment options.”

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EAMS focuses on providing patients with life-threatening or seriously debilitating conditions access to medicines that has not yet received a marketing authorisation when there is a clear unmet medical requirement.

"This decision allows patients with DMD to receive treatment for respiratory function decline who otherwise would not have access to such treatment options."

The MHRA approval enables patients with DMD, who meet criteria defined under this scheme, to gain access to Raxone.

Under the EAMS, Santhera’s Raxone is indicated for slowing the decline of respiratory function in patients with DMD from the age of ten who are currently not taking glucocorticoids.

The decline of respiratory function must be confirmed by carrying out repeated measurements before the initiation of the treatment.

The treatment can be used in patients previously treated with glucocorticoids or in patients in whom glucocorticoid treatment is not tolerated or is considered inadvisable.

Image: Histopathology of gastrocnemius muscle from a patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Photo: courtesy of Dr. Edwin P. Ewing, Jr.