Santhera's Raxone® Receives First Positive EAMS Scientific Opinion from UK's MHRA in Duchenne Muscular Dystrophy
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Santhera Pharmaceuticals Holding AG /
Santhera's Raxone® Receives First Positive EAMS Scientific Opinion from UK's
MHRA in Duchenne Muscular Dystrophy
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The issuer is solely responsible for the content of this announcement.
Liestal, Switzerland, June 22, 2017 - Santhera Pharmaceuticals (SIX: SANN)
announces that the UK's Medicines and Healthcare products Regulatory Agency
(MHRA) has granted Raxone (idebenone) a positive scientific opinion through the
Early Access to Medicines Scheme (EAMS) for patients with respiratory function
decline not taking glucocorticoids in Duchenne Muscular Dystrophy (DMD).
The aim of the EAMS is to provide patients with life threatening or seriously
debilitating conditions access to medicines that do not yet have a marketing
authorization when there is a clear unmet medical need.(1) The MHRA decision
allows patients with DMD, who meet criteria defined under this scheme, to gain
access to Raxone, an investigational medicinal product currently under review
for DMD for Marketing Authorization by the European Medicines Agency (EMA).
Under the EAMS, and as shown in the public assessment report,(2) Raxone is
indicated for slowing the decline of respiratory function in patients with DMD
from the age of 10 years who are currently not taking glucocorticoids. The
decline of respiratory function must be confirmed by repeated measurements prior
to initiation of treatment. Raxone can be used in patients previously treated
with glucocorticoids or in patients in whom glucocorticoid treatment is not
tolerated or is considered inadvisable.
"We're proud to receive the positive EAMS scientific opinion for Raxone in the
UK and to have our lead compound designated as a promising innovative medicine,
the first for a drug intended for the treatment of DMD," said Thomas Meier, PhD,
CEO of Santhera. "This decision allows patients with DMD to receive treatment
for respiratory function decline who otherwise would not have access to such
treatment options."
"This is excellent news for patients with respiratory decline in Duchenne
muscular dystrophy," said Janet Bloor, Chair of the Board of Trustees at Action
Duchenne. "The need for new treatments in DMD is very great and the EAMS can
help to accelerate access for patients. Action Duchenne was pleased to provide
advice during the development of the EAMS program and we are delighted to see
this first positive opinion in DMD."
About the UK Early Access to Medicines Scheme (EAMS)
The UK's industry-sponsored EAMS aims to give patients with life threatening or
seriously debilitating conditions access to medicines that do not yet have a
marketing authorization when there is a clear unmet medical need. Under the
scheme, the MHRA provides a scientific opinion on the benefit/risk balance of
the medicine, based on the data available when the EAMS submission was made. The
opinion lasts for a year and can be renewed. The scheme is voluntary and the
opinion from MHRA does not replace the normal licensing procedures for
medicines.
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(®) (idebenone) 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), Santhera has
filed a Marketing Authorization Application in the European Union and
Switzerland for DMD patients with respiratory function decline who are not
taking glucocorticoids. In collaboration with the U.S. 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 Chris Welsh, Consilium
Strategic Communications
Phone +1 212 915 2568 Phone
+1 646 586 2113
hans(at)lifesciadvisors.com santhera(at)consilium-
comms.com
European Public Relations contact
Amber Fennell, Consilium Strategic Communications
Phone +44 20 3709 5700
santhera(at)consilium-comms.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.
References
(1) Medicines and Healthcare products Regulatory Agency Patient safety and
Marketing authorisations, variations and licensing guidance. Available at:
https://www.gov.uk/guidance/apply-for-the-early-access-to-medicines-scheme-eams
(June 2017)
(2) Public assessment report. Available at
https://www.gov.uk/government/publications/early-access-to-medicines-scheme-
eams-scientific-opinion-raxone-to-treat-the-decline-of-respiratory-function-in-
patients-with-duchenne-muscular-dys
# # #
News Release EAMS:
http://hugin.info/137261/R/2114963/804723.pdf
This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
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: 22.06.2017 - 07:00 Uhr
Sprache: Deutsch
News-ID 549238
Anzahl Zeichen: 7800
contact information:
Town:
Liestal
Kategorie:
Business News
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