Actelion updates US Healthcare Professionals for patients with type 1
Gaucher disease - Information on the potential use of Zavesca®
(miglustat) oral capsule to address the supply shortage of
ALLSCHWIL/BASEL, SWITZERLAND - 8 September 2009 - Actelion Ltd (SIX:
ATLN) has distributed updated information to Healthcare Professionals
who treat type 1 Gaucher patients in the United States regarding the
potential use of Zavesca® (miglustat) to minimize the impact of the
supply shortage of imiglucerase (produced and marketed by Genzyme
Corporation under the brand name Cerezyme®).
Despite strict supply conservation measures currently being
implemented, some patients are facing the prospect of an interruption
of their imiglucerase treatment. This shortage is now expected to
last longer and to affect more patients than initially predicted in
June 2009 by the product manufacturer.
Accordingly, Actelion has taken all the necessary steps to ensure
access to Zavesca® where this important oral therapy has been
approved for use by patients with Type 1 Gaucher disease, as
determined by their physicians. In the United States, Zavesca® has
been available since 2003.
Following communication with the US Food and Drug Administration
(FDA), Actelion has issued the following letter to remind Health Care
Professionals in the United States about the availability of Zavesca®
as an immediate treatment option for appropriate type 1 Gaucher
patients in relation to the current imiglucerase shortage. Health
Care Professionals may also receive additional information by email
(email@example.com) or by calling toll-free (866) 228-3546.
Notes to the editor:
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 (European
Union) or is not a therapeutic option (United States). It is approved
in the European Union, the United States, Canada, Switzerland,
Brazil, Australia, Turkey, Israel, South Korea and New Zealand.
In the European Union, South Korea and Brazil, Zavesca® is also
indicated for the treatment of progressive neurological
manifestations in adult patients and paediatric patients with
Niemann-Pick type C disease.
About type 1 Gaucher disease
Type 1 Gaucher disease, an inherited autosomal recessive disorder, is
the most common disease among all inherited glycosphingolipid storage
disorders, affecting approximately 7,000 patients world-wide. It is a
multi-system disease characterized by hepatomegaly, splenomegaly,
anemia and thrombocytopenia. In addition, bone manifestations are
often underdiagnosed and include osteopenia, lytic lesions, chronic
pain, acute episodes of 'bone crisis', bone infarcts, and
osteonecrosis, resulting in long-term disability. Approved treatments
for patients with type 1 Gaucher disease include intravenous enzyme
replacement therapy with Cerezyme® (imiglucerase) and oral substrate
reduction therapy with Zavesca® (miglustat).
Zavesca® safety information
Gastrointestinal events, mainly diarrhea, have been observed in more
than 80% of patients treated with Zavesca®, either at the onset of
treatment or intermittently during treatment. The majority of cases
are mild and are expected to resolve after the first weeks on
therapy. In clinical practice, diarrhea has been observed to respond
to diet modification (reduction of lactose and other carbohydrate
intake), to taking Zavesca® away from meals, and/or to antidiarrheal
medicinal products such as loperamide. In some patients, temporary
dose reduction may be necessary. Patients with chronic diarrhea or
other persistent gastrointestinal events that do not respond to these
interventions should be investigated according to clinical practice.
Zavesca® has not been evaluated in patients with a history of
significant gastrointestinal disease, including inflammatory bowel
Cases of peripheral neuropathy have been reported in patients with
type 1 Gaucher disease treated with Zavesca®. Peripheral neuropathy
seems to be more common in patients with type 1 Gaucher disease
compared to the general population. All patients should undergo
baseline and repeat neurological evaluation. Patients who develop
symptoms such as numbness and tingling should have a careful
re-assessment of risk benefit.
Zavesca® may cause fetal harm if administered to a pregnant woman and
is contraindicated in women who are or who may become pregnant;
patients should be informed of the potential hazard to the fetus.
There is a risk of impaired fertility in men. Men should maintain
reliable contraceptive methods and not plan to conceive while taking
Zavesca® and for three months thereafter.
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 2000 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:
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
This announcement was originally distributed by Hugin. The issuer is
solely responsible for the content of this announcement.
Copyright © Hugin AS 2009. All rights reserved.
durchschnittliche Punktzahl: 0|