Switzerland-based Santhera Pharmaceuticals has received ‘Autorisation Temporaire d’Utilisation dite de cohorte’ (cohort ATU), a temporary authorisation, from the French National Agency for Medicines and Health Products Safety (ANSM) for the use of Raxone in the treatment of Leber’s Hereditary Optic Neuropathy (LHON).
The company said that the ATU system allows patients in France to receive reimbursed treatment with a product before a marketing authorisation is granted in the EU.
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The temporary authorisation was granted after an assessment by the ANSM and clinical experts of a full application dossier comprising quality, clinical efficacy and safety data, including new efficacy data collected from LHON patients taking part in an ongoing expanded access programme (EAP).
Under the cohort ATU, the company will provide Raxone to LHON patients in France, with government allocation to hospitals to ensure reimbursement, until a full marketing authorisation is granted in the EU.
Santhera CEO Thomas Meier said: “We will work diligently to assist LHON patients in France in gaining access to Raxone as quickly as possible under this programme.
“The revenues from the cohort ATU will assist in off-setting previous development costs for Raxone in this indication.”
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By GlobalDataLHON is a heritable genetic disease that causes blindness and it typically presents in young adults, mostly men, as rapid, painless loss of central vision in one eye, followed by visual loss in the fellow eye within a few months of the onset of symptoms, leading to blindness.
The company intends to file marketing authorisation application (MAA) for Raxone in the EU in the first quarter of 2014.
Image: The blood vessels in a normal human retina. Photo: courtesy of Mikael Häggström.
