Update on CHMP negative opinion for Raxone (idebenone)

This afternoon, Action Duchenne has since had confirmation by the Santhera team and this article that this decision has no consequences on their ongoing programs. Specifically, those who have enrolled in the Early Access to Medicines Scheme to access Raxone or those who are eligible and have enrolled in Santhera’s randomized, double-blind, placebo-controlled phase III (SIDEROS) trial.

The European Medicines Agency published their lay question and answer session on their website with a summary of the reasons behind their negative opinion directly below. A summary to the full article can be found here: http://www.ema.europa.eu/docs/en_GB/document_library/Summary_of_opinion/human/003834/WC500234840.pdf.

Santhera provided results from a main study involving 64 patients with Duchenne muscular dystrophy who were not being treated with corticosteroids. Raxone was compared with placebo (a dummy treatment) and the main measure of effectiveness was the change in PEF (peak expiratory flow, the maximum speed a person can breathe out air) after one year of treatment. PEF is an indicator of breathing function.

The CHMP was of the opinion that the study results provided by the company were insufficient to determine the benefit of Raxone in patients with Duchenne muscular dystrophy. Although a difference in PEF in favour of Raxone was observed, there was no clear improvement in other indicators of breathing function or in muscle strength, motor function or quality of life. The Committee also had some concerns about the way the study was conducted and analysed. Therefore, the CHMP was of the opinion that the benefits of Raxone in patients with Duchenne muscular dystrophy did not outweigh its risks. Hence, the CHMP recommended that the change to the marketing authorisation be refused.

As stated in this morning’s news article, Santhera intends to appeal this opinion and seek re-examination by the CHMP.