Leber’s Hereditary Optic Neuropathy Treament Granted Temporary Authorisation

In Market Access, Press Release, Regulation & Government by CameronLeave a Comment

The French National Agency for Medicines and Health Products Safety (ANSM) yesterday granted temporary authorisation (ATU) to Santhera’s Raxone (Idebenone). Sufferers of Leber’s Hereditary Optic Neuropathy in France will now have access to the treatment under the ATU system. The ATU system allows treatments, not yet authorised by the EU, to be made available to patients where there is a genuine public health need, and a lack of any therapeutic alternative.

The ATU was granted after a positive review from the ANSM of a substantial dossier submitted by the Swiss-based pharmaceutical company, containing up to date quality, clinical efficacy and safety data.

“We are very pleased that the ANSM has positively reviewed the submitted dossier and has taken the step of granting a temporary authorization for use for Raxone® in the treatment of LHON, whilst we continue to work towards a full market approval with the European regulatory authorities” Thomas Meier, CEO of Santhera

Leber’s Hereditary Optic Neuropathy (LHON) is a heritable genetic disease causing blindness. Typically affecting young adults, mostly men, causing painless loss of central vision in one eye followed by the other. Over 95% of LLHON patients have at least one of three pathogenic mutations of the mitochondrial DNA, which eventually leads to decreased cellular energy production.

see the press release here.

 

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