Scottish Medicines Consortium Approves Santhera's Raxone® (idebenone) for Restricted Use in Patients with LHON within NHS Scotland
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Santhera Pharmaceuticals Holding AG /
Scottish Medicines Consortium Approves Santhera's Raxone® (idebenone) for
Restricted Use in Patients with LHON within NHS Scotland
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Liestal, Switzerland, May 9, 2017 - Santhera Pharmaceuticals (SIX: SANN)
announces approval by the Scottish Medicines Consortium for restricted use of
Raxone(®) (idebenone) in patients with LHON, with effect May 8, 2017.
Patients with Leber's hereditary optic neuropathy (LHON), an extremely rare,
inherited cause of blindness, could benefit from the only licensed treatment to
date, following advice issued by the Scottish Medicines Consortium (SMC) under
the ultra-orphan medicine process. The SMC accepted Raxone(®) (idebenone) for
restricted use by NHS Scotland in the treatment of visual impairment in
adolescent and adult patients with LHON who are not yet blind (i.e., they do not
meet the UK criteria to be registered as severely sight impaired). Raxone was
accepted following consideration through SMC's Patient and Clinician Engagement
(PACE) process, for medicines used at the end of life and for very rare
conditions. Scotland is the first country in the UK to make Raxone available for
the treatment of LHON. The SMC advice takes into account the benefits of a
Patient Access Scheme (PAS), which improves the cost-effectiveness of Raxone.
LHON is a devastating disease that causes blindness mainly in young men during
their late teens/early adulthood. Patients suddenly lose central vision and can
no longer see fine details, read print or recognize faces. Within 12 months from
onset, about 80% of untreated patients will have become legally blind. This is
exceptionally debilitating and isolating in young adults, affecting their
education, career plans and overall quality of life. Russell Wheeler, Trustee of
the UK based LHON Society said: "Despite LHON being identified almost 150 years
ago, the lack of any approved treatment for this devastating condition until now
has led to many patients feeling helpless and abandoned by the health service.
We applaud the pragmatism of the SMC's decision following a rigorous and
transparent process. The availability of Raxone to affected families in Scotland
is a welcome step towards avoiding preventable blindness in patients with LHON
and we hope it will be followed by similar availability in other parts of the
UK."
Raxone treatment is now available to LHON patients under different reimbursement
models in several European countries. Giovanni Stropoli, EVP, Chief Commercial
Officer Europe & ROW commented, "We are grateful to the SMC for their
comprehensive evaluation of our dossier for Raxone. As we continue working with
the health services in other parts of the UK we hope that this positive advice
to NHS Scotland will be followed soon by similar availability for LHON patients
across the United Kingdom."
The full SMC advice can be reviewed at the following link:
http://www.scottishmedicines.org.uk/SMC_Advice/Advice/1226_17_idebenone_Raxone/i
debenone_Raxone. Please also refer to the full Summary of Product
Characteristics at: http://www.medicines.org.uk/emc/medicine/32655.
About Leber's Hereditary Optic Neuropathy and the Therapeutic Use of Raxone
LHON is a heritable genetic disease causing blindness. The disease presents
predominantly in young, otherwise healthy adult males as rapid, painless loss of
central vision, usually leading to permanent bilateral blindness within a few
months of the onset of symptoms. About 95% of patients harbor one of three
pathogenic mutations of the mitochondrial DNA, which cause a defect in the
complex I subunit of the mitochondrial respiratory chain. This defect leads to
decreased cellular energy (ATP) production, increased reactive oxygen species
(ROS) production and retinal ganglion cell dysfunction, which cause progressive
loss of visual acuity and blindness.
Raxone (idebenone), a synthetic short-chain benzoquinone and a cofactor for the
enzyme NAD(P)H:quinone oxidoreductase (NQO1), circumvents the complex I defect,
reduces and scavenges ROS, restores cellular energy levels in retinal ganglion
cells and promotes recovery of visual acuity.
Raxone is an oral medication, authorized at a daily dose of 900 mg (given as 2
tablets three times a day with food), for the treatment of visual impairment in
adolescent and adult patients with LHON. Efficacy data come from Santhera's
randomized, placebo-controlled 24-week RHODOS trial, from the open label
Expanded Access Program of 'real-life' usage and from a natural history study of
untreated patients, which together have demonstrated that vision loss can be
mitigated or reversed in patients treated with Raxone. Treatment should be
initiated and supervised by a physician with experience in LHON.
About Santhera
Santhera Pharmaceuticals (SIX: 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.
Santhera's lead product Raxone is authorized in the European Union, Norway,
Iceland and Liechtenstein for the treatment of Leber's hereditary optic
neuropathy (LHON). For Duchenne muscular dystrophy (DMD), the second indication
for Raxone, Santhera has filed a Marketing Authorization Application (MAA) in
the European Union and Switzerland. In collaboration with the US National
Institute of Neurological Disorders and Stroke (NINDS) Santhera is developing
Raxone in a third indication, primary progressive multiple sclerosis (PPMS), and
omigapil for congenital muscular dystrophy (CMD), all areas of high unmet
medical need. For further information, please visit the Company's website
www.santhera.com.
Raxone(®) is a trademark of Santhera Pharmaceuticals.
For further information, contact:
Thomas Meier, PhD, Chief Executive Officer Christoph Rentsch, Chief
Financial Officer
Phone +41 61 906 89 64 Phone
+41 61 906 89 65
thomas.meier(at)santhera.com
christoph.rentsch(at)santhera.com
Sue Schneidhorst, Head Group Communications
Phone +41 61 906 89 26
sue.schneidhorst(at)santhera.com
US investor contact US Public
Relations contact
Hans Vitzthum, LifeSci Advisors, LLC John Gillespie, Medical
Dynamics
Phone +1 212 915 2568 Phone
+1 646 599 8626
hans(at)lifesciadvisors.com
jgillespie(at)rxmedyn.com
Disclaimer / Forward-looking statements
This communication does not constitute an offer or invitation to subscribe for
or purchase any securities of Santhera Pharmaceuticals Holding AG. This
publication may contain certain forward-looking statements concerning the
Company and its business. Such statements involve certain risks, uncertainties
and other factors which could cause the actual results, financial condition,
performance or achievements of the Company to be materially different from those
expressed or implied by such statements. Readers should therefore not place
undue reliance on these statements, particularly not in connection with any
contract or investment decision. The Company disclaims any obligation to update
these forward-looking statements.
# # #
News Release SMC:
http://hugin.info/137261/R/2102790/797484.pdf
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The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Santhera Pharmaceuticals Holding AG via GlobeNewswire
Unternehmensinformation / Kurzprofil:
Datum: 09.05.2017 - 07:00 Uhr
Sprache: Deutsch
News-ID 541039
Anzahl Zeichen: 8915
contact information:
Town:
Liestal
Kategorie:
Business News
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