Raxone®▼(idebenone) recommended for use within NHS Wales for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON)

Raxone^®▼(idebenone) recommended for use within NHS Wales for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON)

• Idebenone is the first licensed medicine available for the treatment of Leber’s Hereditary Optic Neuropathy (LHON) on the NHS in Wales
• Idebenone is now recommended and available on the NHS in Wales, Scotland and Northern Ireland

Manchester, UK, 22 April 2021 – Chiesi Limited is pleased to announce that the All Wales Medicines Strategy Group (AWMSG) has recommended Raxone^®▼ (idebenone) for use within NHS Wales for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON).¹ This recommendation applies only in circumstances where the approved Wales Patient Access Scheme (WPAS) is utilised or where the list/contract price is equivalent or lower than the WPAS price.¹

LHON is a rare genetic eye condition caused by a mutation in mitochondrial DNA that leads to visual impairment. It is is a maternally inherited disease with an estimated prevalence between 1:15,000 to 1:50,000 worldwide.² LHON can occur at any age but mostly affects young men (80–90% of cases) in their second and third decades of life.^3,4,5,6

“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, debiliating disease – the majority of whom are young men - and their families,” said Tom Delahoyde, Managing Director of Chiesi in the UK and Ireland. “Idebenone will address the significant unmet need that exists for people with LHON and it’s recommedation today marks our continued commitment to rare disease communities.”

Idebenone is a short-chain benzoquinone derivative and synthetic analogue of ubiquinone (coenzyme Q10).² It is believed to inhibit lipid peroxidation, protecting cell membranes and mitochondria from oxidative damage.² Idebenone (Raxone) is considered as an ultra-orphan medicine according to the criteria in the AWMSG appraisal process for a medicine for a rare disease. Idebenone is now reimbursed in Wales, Scotland and Northern Ireland, but not yet England.

“We are  extremely pleased that people in Wales with this devastating condition have the opportunity to access idebenone,” said Alessio Bastianello, Head of Rare Disease Cluster Central-North EU. “Chiesi is committed to discovering, developing and commercialising innovative therapies to address unmet needs for people living with rare diseases. We look forward to working with NHS England to make idebenone available across England.”