Chiesi’s rare disease medicine Raxone has been recommended for use within NHS Wales for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON).
LHON, a maternally inherited disease, is caused by a mutation in mitochondrial DNA that results in visual impairment. Although it can occur at any age, LHON commonly affects young men in their 20s and 30s.
Raxone (idebenone) is believed to inhibit lipid peroxidation, protecting cell membranes and mitochondria from oxidative damage.
According to the criteria in the All Wales Medicines Strategy Group (AWMSG) appraisal process, Raxone is considered an ultra-orphan medicine.
The AWMSG recommendation for Raxone only applies when the approved Wales Patient Access Scheme (WPAS) is used or where the list price is equivalent or lower than the WPAS price.
Currently, Raxone is now reimbursed for use within the NHS in Wales, Scotland and Northern Ireland.
“There are currently no other licensed medicines available for the treatment of LHON on the NHS in Wales and current clinical practice is limited to best supportive care. So this is a potentially life-changing milestone for people in Wales who have been diagnosed with this ultra-rare, debilitating disease – the majority of whom are young men – and their families,” said Tom Delahoyde, managing director of Chiesi in the UK and Ireland.
“[Raxone] will address the significant unmet need that exists for people with LHON and its recommendation today marks our continued commitment to rare disease communities,” he added