First Patients Enrolled in UK's Early Access to Medicines Scheme for Santhera's Raxone® in Duchenne Muscular Dystrophy (DMD)
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Santhera Pharmaceuticals Holding AG /
First Patients Enrolled in UK's Early Access to Medicines Scheme for Santhera's
Raxone® in Duchenne Muscular Dystrophy (DMD)
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The issuer is solely responsible for the content of this announcement.
Liestal, Switzerland, September 11, 2017 - Santhera Pharmaceuticals (SIX: SANN)
announces enrollment of first DMD patients with respiratory function decline not
taking glucocorticoids into UK's Early Access to Medicines Scheme (EAMS) for
Raxone(®).
In June, Raxone(®) (idebenone) was granted a positive scientific opinion through
the Early Access to Medicines Scheme (EAMS) by the UK's Medicines and Healthcare
products Regulatory Agency (MHRA) - the first drug approved under the EAMS for
patients with DMD. 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 Marketing Authorization in DMD by
the European Medicines Agency (EMA). Enrollment into the EAMS documents the
first use of Raxone outside of a clinical trial for patients with DMD.
"I am pleased to be able to offer Raxone to several of my patients in
respiratory decline, as there are no other medical treatments available," said
Dr. Dipansu Ghosh, a respiratory physician at a DMD center based in Leeds.
To date, 15 specialist DMD centers in the UK have received training under the
requirements of the EAMS. Several additional sites have expressed an interest to
be trained and are currently undergoing local approval processes.
"At Action Duchenne we were encouraged by the positive EAMS opinion, earlier
this summer. Particularly, for young people living with Duchenne who have
respiratory decline," said Janet Bloor, Chair of Trustees of Action Duchenne. "I
am delighted that respiratory function is being considered by the regulatory
agencies. This will hopefully pave the way for more potential treatments that
may benefit the wider spectrum of DMD patients."
Under the EAMS, and as shown in the public assessment report,( 1) 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.
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, Liechtenstein and Israel 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:
Sue Schneidhorst, Head Group Communications
Europe: +41 61 906 89 26
US: +1 646 586 2113
sue.schneidhorst(at)santhera.com
Investors:
Christoph Rentsch, Chief Financial Officer Hans Vitzthum,
LifeSci Advisors
Europe: +41 61 906 89 65 US:
+1 212 915 2568
christoph.rentsch(at)santhera.com
hans(at)lifesciadvisors.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.
Reference
(1) Public EAMS 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 Enrollment:
http://hugin.info/137261/R/2132829/815304.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: 11.09.2017 - 07:00 Uhr
Sprache: Deutsch
News-ID 559350
Anzahl Zeichen: 7033
contact information:
Town:
Liestal
Kategorie:
Business News
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