Please note there are currently no clinical trials for LHON planned to start but we will put information on the Newcastle University website if that situation changes.
At this moment in time, Idebenone, available under the brand name Catena® has only got a marketing licence in Canada for the treatment of Friedreich's Ataxia, a hereditary neurodegenerative condition. Santhera Pharmaceuticals, the holder of the marketing licence in Canada, has also conducted clinical trials to test the safety and efficacy of Idebenone in LHON and in other indications. The promising results of the RHODOS study (SNT-II-003) combined with the urgent unmet medical need for LHON made Santhera take the decision to submit a Marketing Authorization Application to the European Medicines Agency EMA. This submission is currently under review by the EMA. Provided that no undue stops will affect the review process, EMA's decision may be expected towards the end of 2012. In case of a positive decision, Santhera will then be able to start the launch process in individual EU countries. (Please note that in this case, the brand name will be different from Catena.)
In order to make Catena available to patients before it has been formally licensed Santhera is making use of the possibility provided for by EC Directive 2001/83/ec , Art. 5, paragraph 1 which says: "A Member State may, in accordance with legislation in force and to fulfil special needs, exclude from the provisions of this Directive (Santhera: i.e. the requirement of a Marketing Authorization) medicinal products supplied in response to a bona fide unsolicited order, formulated in accordance with the specifications of an authorised health-care professional and for use by an individual patient under this direct personal responsibility."
Santhera has set up a Named Patient Programme to distribute Catena to patients who have got a licensed doctor's prescription and who - through their doctor or a pharmacist - have placed an order to the distributor contracted by Santhera. For information call 00 41 61 906 89 50 or email: firstname.lastname@example.org
RHODOS, a randomized, double-blind, placebo-controlled clinical trial which included 85 patients and had a treatment duration of 24 weeks showed that patients with discordant visual acuity were the most likely to benefit from a treatment with Idebenone. "Discordant" meaning that the visual acuity was different between the two eyes of a patient, which is usually observed in early stages of the disease. On the other side, the treatment with Idebenone as opposed to the treatment with placebo did show an improvement in 20% of the eyes of patients who were not able to read any letter on the chart at the beginning of the study. The patients in this group, if they showed an improvement at all, were able to read at least one row of letters in the chart by the end of the study. Based on the available data it is, however, not possible to make any statement on the probability whether you personally could benefit from Idebenone treatment. The study also showed that patients with the most common disease-causing mtDNA mutations in Europe (the so called “11778” and “3460” mutations) are more likely to benefit from idebenone. The treatment effect was less obvious in patients with the 14484 mutation, as there is already a comparably high spontaneous vision recovery in these patients.
The findings of the RHODOS trial indicated that the 900mg daily dose of Idebenone was safe and well tolerated.
Idebenone, with the brand name of Catena®, has currently got a marketing licence only in Canada for the treatment of Friedreich's Ataxia. It has not got a marketing authorization for the treatment of LHON in any country. However, through the Named Patient Programme offered by Santhera Pharmaceuticals, Catena can be made available. Details contained above.
In general, data from the RHODOS study show that patients who were at risk of further vision loss (defined as patients with discordant visual acuity in both eyes) were protected from further vision loss. There was also some indication that a proportion of patients regained visual acuity (e.g. from hand motion to being able to read at least 1 full line on the letter chart). However, it has to be borne in mind that the onset of visual loss in participants in the RHODOS study has been five years or less at the beginning of the study. RHODOS does not deliver data which allow to assume a similar effect in patients like you who have been affected with the disease much longer.
With thanks to Santhera Pharmaceuticals for information contained in this article.
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