Scottish Medicines Consortium Approves Santhera's Raxone® (idebenone) for Restricted Use in Patients with LHON within NHS Scotland - Seite 2
Raxone treatment is now available to LHON patients under different reimbursement models in several European countries. Giovanni Stropoli, EVP, Chief Commercial Officer Europe & ROW commented, "We are grateful to the SMC for their comprehensive evaluation of our dossier for Raxone. As we continue working with the health services in other parts of the UK we hope that this positive advice to NHS Scotland will be followed soon by similar availability for LHON patients across the United Kingdom."
The full SMC advice can be reviewed at the following link: http://www.scottishmedicines.org.uk/SMC_Advice/Advice/1226_17_idebenon .... Please also refer to the full Summary of Product Characteristics at: http://www.medicines.org.uk/emc/medicine/32655.
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About Leber's Hereditary Optic Neuropathy and the Therapeutic Use of Raxone
LHON is a heritable genetic disease causing blindness. The disease presents predominantly in young, otherwise healthy adult males as rapid, painless loss of central vision, usually leading to
permanent bilateral blindness within a few months of the onset of symptoms. About 95% of patients harbor one of three pathogenic mutations of the mitochondrial DNA, which cause a defect in the
complex I subunit of the mitochondrial respiratory chain. This defect leads to decreased cellular energy (ATP) production, increased reactive oxygen species (ROS) production and retinal
ganglion cell dysfunction, which cause progressive loss of visual acuity and blindness.
Raxone (idebenone), a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1), circumvents the complex I defect, reduces and scavenges ROS,
restores cellular energy levels in retinal ganglion cells and promotes recovery of visual acuity.
Raxone is an oral medication, authorized at a daily dose of 900 mg (given as 2 tablets three times a day with food), for the treatment of visual impairment in adolescent and adult patients
with LHON. Efficacy data come from Santhera's randomized, placebo-controlled 24-week RHODOS trial, from the open label Expanded Access Program of 'real-life' usage and from a natural history study
of untreated patients, which together have demonstrated that vision loss can be mitigated or reversed in patients treated with Raxone. Treatment should be initiated and supervised by a physician
with experience in LHON.