drugs

Raxone - Idebenone

What is Raxone and Idebenone used for and what is it used for?

Raxone is a medicine indicated for the treatment of visual impairment in adults and adolescents from 12 years of age with Leber's hereditary optic neuropathy (LHON), an inherited disorder characterized by progressive loss of vision. 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 Raxone is similar to a "reference medicine" that contains the same active ingredient, but with a different formulation. The reference medicine for Raxone is Mnesis (45 mg tablets).

How is Raxone - Idebenone used?

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

How does Raxone - Idebenone work?

The active ingredient in Raxone, idebenone, is an antioxidant agent that acts on the mitochondria (the cellular structures that produce the energy necessary for the functioning of the cells). Mutations (defects) of the mitochondria genetic material are observed in patients with Leber's hereditary optic neuropathy. This means that the mitochondria do not function properly and, instead of generating energy, produce toxic forms of oxygen (free radicals) that damage the eye's nerve cells responsible for vision. Idebenone is believed to help improve energy production by restoring mitochondrial function, so as to prevent the cellular damage and vision loss observed in LHON.

What benefit has Raxone - Idebenone shown during the studies?

Raxone was studied in one main study involving 85 patients with Leber's hereditary optic neuropathy, in which Raxone was compared with placebo (a dummy treatment) over a period of 24 weeks. The main measure of effectiveness was the improvement of eyesight, recorded mostly on the number of letters that patients were able to read when examining the standard octotype view. At the end of the study, patients treated with Raxone were able to read on average 3-6 more letters than those on placebo. Furthermore, some patients who at the beginning of the study were unable to read the optotypical table (classified as "off chart") were able to read at least one line at the end of treatment, and this was another clinically important result. Furthermore, in 30% of subjects treated with Raxone (16 of 53) a clinically significant recovery of vision was observed in at least one eye compared to 10% of patients (3 of 29) in the placebo group.

Additional data supporting the benefits of Raxone were taken from an extended access program, in which Raxone was made available to individual patients not participating in a clinical study, as well as a study case survey, which included data of patients with LHON not undergoing any therapy.

From the analysis of all these data a coherent model emerged that, in general, a greater percentage of patients treated with Raxone showed improvements in their vision compared to patients who were not treated or treated with placebo.

What is the risk associated with Raxone - Idebenone?

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

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

Why has Raxone - Idebenone been approved?

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

The committee noted the absence of therapies for the prevention or treatment of visual impairment in patients with LHON. The results of the main study showed improved vision in patients treated with Raxone and this trend towards a beneficial effect was confirmed by additional data from an extended access program and a case study survey.

Regarding the safety of Raxone, the side effects seen with the medicine were mostly mild or moderate in intensity.

Raxone was authorized in "exceptional circumstances" because complete information on Raxone could not be obtained due to the rarity of the disease. Every year the European Medicines Agency will review the new information available and this summary will be updated accordingly.

What information is still awaited for Raxone - Idebenone?

As Raxone has been authorized in exceptional circumstances, the company that markets Raxone will carry out further studies on the long-term effects and safety of the medicine, and will establish and maintain a register of patients with LHON treated with the medicine.

What measures are being taken to ensure the safe and effective use of Raxone - Idebenone?

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.

More information on Raxone - Idebenone

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

The summary of the opinion of the Raxone Committee for Orphan Medicinal Products is available on the Agency's website: ema.Europa.eu/Find medicine / Human medicines / Rare disease designation.