Gaucher Community News - National Gaucher Foundation

More Patients on
Zavesca® (miglustat) in 2009
Submitted by Ashley Smith
Actelion Pharmaceuticals
This has been a busy year for the Gaucher
community. At Actelion, we are continuing
our efforts to provide patients with treatment
options to better manage their disease today and
simultaneously pursuing important research to
develop even better therapies for patients in the
future.
We recognize that this year has brought challenges
to many patients living with Type 1 Gaucher
disease, due to the imiglucerase shortage. During
2009, we significantly ramped up production of
Zavesca® (miglustat) due to increased interest in
Zavesca by health care professionals and we have
seen an increase in the number of patients taking
Zavesca.
Advancing Treatment through Ongoing Research
Actelion is committed to following scientific
innovation where it leads, and we constantly drive
ourselves to learn more about Type 1 Gaucher
disease and lysosomal storage disorders to provide
better treatments for patients.
Actelion has been busy advancing an ongoing study
this year which looks at the safety and efficacy
of Zavesca at 24-months in patients with Type 1
Gaucher. The study completed enrollment last
year, and we expect results from the study in 2010.
We are excited that the FDA has recently
announced a public advisory committee meeting
to review the use of Zavesca for the treatment of
another rare lysosomal storage disorder.
We are also ramping up a number of new studies
which will be run by physicians interested in
learning more about Zavesca’s effects in lysosomal
storage disorders. We believe we will gain valuable
information about patients’ responses to Zavesca
and continue to learn more about the disease, which
could help physicians provide better treatment to
patients.
Sharing Patient Stories
One of our main company goals is to help connect the
Type 1 Gaucher community. As we move into 2010,
we are planning to launch a series of videos of patients
discussing their experiences living with Type 1 Gaucher,
and we hope that these videos will be of value to both
newly-diagnosed and those looking for support from
patients who may be going through similar challenges.
If you are interested in sharing your story about
Type 1 Gaucher disorder or treatment with Zavesca,
please contact Ashley Smith at (650) 243-2368.
Wishing the Gaucher Community a Happy Holiday
Actelion would like to wish the Gaucher community
health and happiness this holiday season and throughout
the next year. We look forward to continuing our efforts
to advance the science and treatment of lysosomal
storage disorders.
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If you are taking Zavesca and/or have questions about
Zavesca, financial support, or access to therapy, contact
your healthcare provider or call Actelion Patient Services at
866-ACTELION (866-228-3546).
Zavesca Indication and Important Safety Information
ZAVESCA is indicated for the treatment of adult patients with
mild to moderate Type 1 Gaucher disease for whom enzyme
replacement therapy is not a therapeutic option
(e.g., due to constraints such as allergy, hypersensitivity,
or poor venous access).
Please see www.ZAVESCA.com for full prescribing information.
Important Safety Information
In clinical studies, the most common adverse events due to
Zavesca included weight loss, diarrhea, and trembling in the
hand. Other common adverse reactions were excess gas,
abdominal pain, headache, and influenza-like symptoms. The
most common serious adverse reaction was muscle weakness
with or without pain and numbness. Patients should undergo
neurological examination at the start of treatment and every
6 months thereafter; Zavesca should be reassessed in patients
who develop symptoms of muscle weakness. Zavesca may
cause fetal harm if administered to a pregnant woman. Men
should maintain reliable contraceptive methods and not plan
to conceive while taking Zavesca and for 3 months after
discontinuing treatment.
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