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Naglazyme® (galsulfase)
for MPS VI
Naglazyme® (galsulfase)
is an enzyme replacement therapy for the treatment
of mucopolysaccharidosis VI (MPS VI), an inherited
life-threatening lysosomal storage disorder caused
by a deficiency of the lysosomal enzyme N-acetylgalactosamine
4-sulfatase.* Naglazyme provides a recombinant version
of this enzyme to individuals diagnosed with MPS
VI.
| Naglazyme
at a Glance |
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Approved
in the United States and European Union
for the
treatment of MPS VI
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Designated
an orphan drug in the United States
and European Union
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Manufactured and
commercialized by BioMarin |
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Naglazyme is approved in both the United States
and the European Union for the treatment of MPS
VI. The U.S. Food and Drug Administration (FDA)
approved the therapy on May 31, 2005 and subsequently,
the European Commission (EC) approved Naglazyme
on January 24, 2006. As the first drug approved
for MPS VI, the FDA and EC have both designated
Naglazyme as an orphan drug, conferring seven years
of market exclusivity in the United States and 10
years of market exclusivity in the European Union.
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BioMarin is commercializing Naglazyme in the United
States and European
Union.
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Full Indication and Important
Safety Information |
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*Naglazyme is indicated for
patients with mucopolysaccharidosis VI (MPS VI).
Naglazyme has been shown to improve walking and
stair-climbing capacity.
The most common adverse events
observed in clinical trials in Naglazyme-treated
patients were headache, fever, arthralgia, vomiting,
upper respiratory infections, abdominal pain, diarrhea,
ear pain, cough, and otitis media. Severe reactions
included angioneurotic edema, hypotension, dyspnea,
bronchospasm, respiratory distress, apnea, and urticaria.
The most common symptoms of infusion reactions included
fever, chills/rigors, headache, rash, and mild to
moderate urticaria. Nausea, vomiting, elevated blood
pressure, retrosternal pain, abdominal pain, malaise,
and joint pain were also reported. No patients discontinued
Naglazyme infusions for adverse events and all patients
that completed the double-blind portion of the trial
continue to receive weekly infusions of Naglazyme.
Nearly all patients developed antibodies as a result
of treatment, but the level of the immune response
did not correlate with the severity of adverse events
or impact the improvements experienced in endurance.
Because antihistamine use may increase the risk
of apneic episodes, evaluation of airway patency
should be considered prior to the initiation of
treatment. Consideration to delay Naglazyme infusion
should be given when treating patients who present
with an acute febrile or respiratory illness. |
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