Japan's Ministry of Health, Labour and Welfare grants approval for Actelion's miglustat for the treatment of Niemann-Pick type C disease
(Thomson Reuters ONE) -
Actelion Pharmaceuticals Ltd /
Japan's Ministry of Health, Labour and Welfare grants approval for Actelion's
miglustat for the treatment of Niemann-Pick type C disease
. Processed and transmitted by Thomson Reuters ONE.
The issuer is solely responsible for the content of this announcement.
ALLSCHWIL/BASEL, SWITZERLAND - 02 April 2012 - Actelion Ltd (SIX: ATLN)
announced today that Japan's Ministry of Health, Labour and Welfare has granted
approval for miglustat for the treatment of Niemann-Pick type C disease.
The approval was based on the data generated for the first approval of miglustat
in this indication in the European Union, as well as data specifically generated
for the submission to the Japanese Health Authorities.
Satoshi Tanaka, President of Actelion Japan and South Korea commented: "I am
very proud to have achieved this successful approval. Our organization has been
able to generate the additional clinical data required for the first approval of
miglustat in Japan, successfully registered the drug in this orphan disease and
we are looking forward to bringing the first therapy to patients with this
devastating neurodegenerative disease."
Actelion Pharmaceuticals Japan will now ensure that miglustat, marketed under
the tradename Brazaves®, is made available to patients as soon as possible.
###
Notes to the editor
About Niemann-Pick type C disease
NP-C disease is a very rare, fatal, neurodegenerative, genetic condition,
primarily affecting children and teenagers although the clinical manifestations
can become apparent at any age. The symptoms are caused by the storage of some
lipids, such as glycosphingolipids and cholesterol, within certain tissues in
the body, including the brain. It is invariably progressive and most patients
die within five to ten years of diagnosis; for the majority, the disease is
fatal during childhood. Neurological deterioration is the key feature of the
disease, and can manifest itself as clumsy body movements, balance problems,
slow and slurred speech, difficulty in swallowing, problems with eye movements
and seizures. Intellectual decline is also common. In the final stages of the
disease the child or young adult is frequently bedridden, has little muscle
control and is intellectually impaired. Diagnosis of the disease can be
difficult and may take years due to the rarity and heterogeneity of this
condition.
About Miglustat
Miglustat, an orally available molecule with a large volume of distribution, is
currently marketed globally under the trade name Zavesca(®).
Zavesca (100 mg miglustat capsule) is indicated for the oral treatment of adult
patients with mild to moderate type 1 Gaucher disease. Zavesca may only be used
in the treatment of type 1 Gaucher patients for whom enzyme replacement therapy
is unsuitable or is not a therapeutic option. It is approved for this indication
in 43 countries.
Zavesca is also indicated for the treatment of progressive neurological
manifestations in adult patients and pediatric patients with Niemann-Pick type C
disease. It is approved for this indication in 42 countries.
Miglustat will be marketed in Japan under the tradename Brazaves®.
Miglustat safety information
In clinical studies, the most common adverse events due to miglustat included
weight loss, diarrhea, and tremor. Other common adverse reactions were
flatulence, abdominal pain, headache, and influenza-like symptoms. The most
common serious adverse reaction was peripheral neuropathy. Patients should
undergo neurological examination at the start of treatment and every 6 months
thereafter; miglustat should be reassessed in patients who develop symptoms of
peripheral neuropathy. Miglustat should not be used in pregnant woman. Men
should maintain reliable contraceptive methods while taking miglustat and for 3
months after discontinuing treatment.
About Actelion Pharmaceuticals Japan
Actelion Pharmaceuticals Japan was established in 2001 to meet the medical needs
of patients with pulmonary arterial hypertension in Japan. Since its foundation
the company has established a significant presence in the pharmaceutical market
and also provides clinical development for the specific needs of the Japanese
Health Authorities. The organization also has a central role for East Asian
cross-border clinical development activities, such as in South Korea. In 2011
Actelion Pharmaceuticals Japan contributed 10% of Actelion's total product
sales.
Actelion Ltd
Actelion Ltd is a biopharmaceutical company with its corporate headquarters in
Allschwil/Basel, Switzerland. Actelion's first drug Tracleer®, an orally
available dual endothelin receptor antagonist, has been approved as a therapy
for pulmonary arterial hypertension. Actelion markets Tracleer® through its own
subsidiaries in key markets worldwide, including the United States (based in
South San Francisco), the European Union, Japan, Canada, Australia and
Switzerland. Actelion, founded in late 1997, is a leading player in innovative
science related to the endothelium - the single layer of cells separating every
blood vessel from the blood stream. Actelion's over 2500 employees focus on the
discovery, development and marketing of innovative drugs for significant unmet
medical needs. Actelion shares are traded on the SIX Swiss Exchange (ticker
symbol: ATLN) as part of the Swiss blue-chip index SMI (Swiss Market Index
SMI®).
For further information please contact:
Roland Haefeli
Senior Vice President, Head of Investor Relations & Public Affairs
Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil
+41 61 565 62 62
+1 650 624 69 36
http://www.actelion.com
Press Release PDF:
http://hugin.info/131801/R/1598822/504269.pdf
This announcement is distributed by Thomson Reuters on behalf of
Thomson Reuters clients. The owner of this announcement warrants that:
(i) the releases contained herein are protected by copyright and
other applicable laws; and
(ii) they are solely responsible for the content, accuracy and
originality of the information contained therein.
Source: Actelion Pharmaceuticals Ltd via Thomson Reuters ONE
[HUG#1598822]
Unternehmensinformation / Kurzprofil:
Datum: 02.04.2012 - 07:02 Uhr
Sprache: Deutsch
News-ID 130702
Anzahl Zeichen: 7183
contact information:
Town:
Allschwil
Kategorie:
Business News
Diese Pressemitteilung wurde bisher 228 mal aufgerufen.
Die Pressemitteilung mit dem Titel:
"Japan's Ministry of Health, Labour and Welfare grants approval for Actelion's miglustat for the treatment of Niemann-Pick type C disease"
steht unter der journalistisch-redaktionellen Verantwortung von
Actelion Pharmaceuticals Ltd (Nachricht senden)
Beachten Sie bitte die weiteren Informationen zum Haftungsauschluß (gemäß TMG - TeleMedianGesetz) und dem Datenschutz (gemäß der DSGVO).
