Temporary Authorization for Use Granted for Santhera's RaxoneÂ® for the Treatment of Leber's Hereditary Optic Neuropathy (LHON) in France
Santhera Pharmaceuticals Holding AG /
Temporary Authorization for Use Granted for Santhera's RaxoneÂ® for the Treatment
of Leber's Hereditary Optic Neuropathy (LHON) in France
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Liestal, Switzerland, JanuaryÂ 21, 2014 - Santhera Pharmaceuticals (SIX:Â SANN)
announced today that the French National Agency for Medicines and Health
Products Safety (ANSM) has granted an "Autorisation Temporaire d'Utilisation
dite de cohorte" (cohort ATU), a Temporary Authorization for Use for Raxone(Â®),
in the treatment of Leber's Hereditary Optic Neuropathy (LHON). The ATU system
allows patients in France to receive reimbursed treatment with a product before
a marketing authorization is granted in the European Union.
Promising treatments that have not yet been granted a marketing authorization
but where the benefit/risk balance is presumed to be positive, based on the
submitted dossier, can be granted a cohort ATU where there is a genuine public
health need in the absence of any suitable therapeutic alternative.
The temporary authorization for Raxone(Â®) 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 participating in an ongoing Expanded Access Program. Santhera will
provide Raxone(Â®)( Â )to LHON patients in France under the cohort ATU, for which
government allocation to hospitals ensures reimbursement, until a full marketing
authorization is granted in the EU.
"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(Â®) in the treatment of LHON, whilst we continue to work towards a full
market approval with the European regulatory authorities", commented Thomas
Meier, CEO of Santhera. "We will work diligently to assist LHON patients in
France in gaining access to Raxone(Â® )as quickly as possible under this program.
The revenues from the cohort ATU will assist in off-setting previous development
costs for Raxone(Â®) in this indication."
About Leber's Hereditary Optic Neuropathy and the use of Raxone(Â®)
Leber's Hereditary Optic Neuropathy (LHON) is a heritable genetic disease
causing blindness. The disease 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. Over 95% of patients harbor one of three pathogenic mutations of the
mitochondrial DNA which cause a defect in the complexÂ I subunit of the
mitochondrial respiratory chain. This defect leads to decreased cellular energy
(ATP) production, increased oxidative stress and retinal ganglion cell
dysfunction which cause progressive loss of visual acuity and blindness.
Raxone(Â®) (idebenone), a synthetic short-chain benzoquinone and a cofactor for
the enzyme NAD(P)H:quinone oxidoreductase (NQO1), is capable of transferring
electrons directly onto complex III of the mitochondrial electron transport
chain, thereby circumventing the complexÂ I defect and restoring cellular energy
levels in retinal ganglion cells and promoting recovery of visual acuity.
Santhera plans to submit a marketing authorization application (MAA) for
Raxone(Â®) in the European Union in the first quarter of 2014.
* * *
Santhera Pharmaceuticals (SIX:Â SANN) is a Swiss specialty pharmaceutical company
focused on the development and commercialization of innovative pharmaceutical
products for the treatment of orphan mitochondrial and neuromuscular diseases,
areas of high unmet medical need with no current therapies. For further
information, please visit www.santhera.com.
Raxone(Â®) is a trademark of Santhera Pharmaceuticals.
For further information, contact
Thomas Meier, Chief Executive Officer
Phone: +41 61 906 89 64
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