Raxone | European Medicines Agency (EMA) (2024)

Application under evaluation
CHMP opinion
European Commission decision

Overview

This is a summary of the European public assessment report (EPAR) for Raxone. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Raxone.

For practical information about using Raxone, patients should read the package leaflet or contact their doctor or pharmacist.

Raxone is a medicine used to treat visual impairment in adults and adolescents aged 12 years and over with Leber’s hereditary optic neuropathy (LHON), an inherited disease characterised by progressive loss of sight. Raxone contains the active substance idebenone.

Because the number of patients with Leber's hereditary optic neuropathy is low, the disease is considered ‘rare’, and Raxone was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 15 February 2007.

Raxone is a ‘hybrid medicine’. This means that it is similar to a ‘reference medicine’ containing the same active substance, but Raxone contains idebenone at a different strength. The reference medicine for Raxone is Mnesis (45 mg tablets).

Raxone can only be obtained with a prescription and treatment should be started and supervised by a doctor with experience in LHON. Raxone is available as 150 mg tablets, and the recommended dose is two tablets taken three times a day with food.

The active substance in Raxone, idebenone, is an anti-oxidant agent that acts on mitochondria (the structures inside cells that produce the energy necessary for cells to function). Patients affected by LHON have mutations (defects) in the genetic material of mitochondria. This means that mitochondria do not work properly to generate energy, and produce toxic forms of oxygen (free radicals) that damage nerve cells in the eye that are needed for vision. Idebenone is thought to help improve production of energy by restoring mitochondrial function, thereby preventing the cellular damage and the loss of sight seen in LHON.

Raxone has been investigated in one main study involving 85 patients with LHON, in which it was compared with placebo (a dummy treatment) over 24 weeks. The main measure of effectiveness was improvement in vision, mostly based on the numbers of letters patients were able to read on a standard eye test chart. By the end of the study, patients treated with Raxone were able to read on average 3 to 6 letters more compared with patients receiving placebo. Furthermore, some patients who were classified as ‘off chart’ (unable to read any letters on the chart) at the beginning of the study were able to read at least one line during the eye test after treatment, and this was also considered clinically important. Additionally, 30% of patients treated with Raxone (16 out of 53) had a clinically relevant recovery of vision in at least one eye, compared with 10% of patients (3 out of 29) in the placebo group.

Additional supportive data on the benefits of Raxone came from an expanded access program through which Raxone was made available to individual patients not participating in a clinical study, and from a case record survey, which included data from patients with LHON who did not receive any treatment.

Analyses of all these data showed a consistent pattern whereby generally a larger proportion of patients treated with Raxone had vision improvement compared with untreated or placebo-treated patients.

The most common side effects with Raxone (which may affect more than 1 in 10 people) are nasopharyngitis and cough; mild to moderate diarrhoea and back pain are also common (affecting up to 1 in 10 people).

For the full list of all side effects and restrictions with Raxone, see the package leaflet.

The Agency’s Committee for Medicinal Products for Human Use (CHMP) decided that the benefits of Raxone are greater than its risks and recommended that it be approved for use in the EU.

The Committee noted the lack of treatments for preventing or reversing the vision loss in patients with LHON. The results of the main study showed an improvement in vision in patients treated with Raxone, and this trend towards a beneficial effect was confirmed by additional data from an expanded access program and a case record survey. With regard to the safety of Raxone, the majority of side effects seen with the medicine were mild or moderate in intensity.

Raxone has been authorised under ‘exceptional circ*mstances’. This is because it has not been possible to obtain complete information about Raxone due to the rarity of the disease. Every year, the European Medicines Agency will review any new information that becomes available and this summary will be updated as necessary.

Since Raxone has been approved under exceptional circ*mstances, the company that markets Raxone will conduct additional studies on the long-term effects and safety of Raxone, and will establish and maintain a registry of LHON patients treated with Raxone.

A risk management plan has been developed to ensure that Raxone is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Raxone, including the appropriate precautions to be followed by healthcare professionals and patients.

Further information can be found in the .

The European Commission granted a marketing authorisation valid throughout the European Union for Raxone on 8 September 2015.

For more information about treatment with Raxone, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

Raxone : EPAR - Summary for the public

Reference Number: EMA/452944/2015

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Product information

Raxone : EPAR - Product Information

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Latest procedure affecting product information: T/0034

19/10/2023

This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.

Product information documents contain:

summary of product characteristics(annex I);
manufacturing authorisationholder responsible for batch release (annex IIA);
conditions of themarketing authorisation(annex IIB);
labelling(annex IIIA);
package leaflet(annex IIIB).

Raxone : EPAR - All Authorised presentations

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Product details

Name of medicine: Raxone
Active substance: idebenone
International non-proprietary name (INN) or common name: idebenone
Therapeutic area (MeSH): Optic Atrophy, Hereditary, Leber
Anatomical therapeutic chemical (ATC) code: N06BX13

Pharmacotherapeutic group

Psychoanaleptics
Other psychostimulants and nootropics

Therapeutic indication

Raxone is indicated for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON).

Authorisation details

EMA product number: EMEA/H/C/003834
Additional monitoring: This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.
Exceptional circ*mstances: This medicine was authorised under exceptional circ*mstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance.
Orphan: This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.
Marketing authorisation holder: Chiesi Farmaceutici S.p.A
Via Palermo 26/A
43122 Parma
Italy
Opinion adopted: 25/06/2015
Marketing authorisation issued: 08/09/2015
Revision: 9

Assessment history

Raxone : EPAR - Procedural steps taken and scientific information after authorisation

English (EN) (185.96 KB - PDF)

First published: 26/04/2018Last updated: 30/10/2023

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Questions and answers on the refusal of a change to the marketing authorisation for Raxone (idebenone)

AdoptedReference Number: EMA/613204/2017

English (EN) (91.49 KB - PDF)

First published: 15/09/2017Last updated: 26/01/2017

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Raxone : EPAR - Public assessment report

AdoptedReference Number: EMA/480039/2015

English (EN) (3.53 MB - PDF)

First published: 17/09/2015Last updated: 17/09/2015

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CHMP summary of positive opinion for Raxone

AdoptedReference Number: EMA/CHMP/368035/2015

English (EN) (70.31 KB - PDF)

First published: 26/06/2015Last updated: 26/06/2015

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News on Raxone

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 January 2018

26/01/2018

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 9-12 October 2017

13/10/2017

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 11 to 14 September 2017

15/09/2017

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 June 2015

26/06/2015

More information on Raxone

EU/3/07/434 - orphan designation for treatment of Leber's hereditary optic neuropathy

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Medicines

This page was last updated on 30/10/2023