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velaglucerase

Velaglucerase alfa is an enzyme replacement therapy used to treat Gaucher disease, a lysosomal storage disorder caused by deficient activity of the enzyme glucocerebrosidase (GCase). It is a recombinant form of human GCase produced in mammalian cell culture and is marketed under the brand name Vpriv. The therapy is designed to supplement the deficient enzyme in patients with Gaucher disease.

Medical uses and mechanism

Velaglucerase alfa is indicated for adults and pediatric patients with confirmed Gaucher disease to help manage

Administration and dosing

The medication is given by intravenous infusion approximately every two weeks. Dosing is weight-based and tailored

Safety and regulatory status

Common adverse effects include infusion-related reactions (fever, chills, flushing), headache, abdominal pain, and rash. Hypersensitivity reactions

History

Velaglucerase alfa was developed for Gaucher disease and received regulatory approval in the 2010s. It is one

manifestations
such
as
anemia,
thrombocytopenia,
hepatosplenomegaly,
and
bone
disease.
As
an
enzyme
replacement
therapy,
it
provides
functional
GCase
to
degrade
glucocerebroside
within
lysosomes,
reducing
the
burden
of
lipid-laden
macrophages
and
improving
hematologic
and
visceral
symptoms.
to
the
individual
patient,
with
infusion
times
typically
ranging
from
about
one
to
several
hours
depending
on
the
dose
and
regimen.
It
is
not
intended
for
neuronopathic
forms
of
Gaucher
disease
where
central
nervous
system
involvement
is
present.
and
the
potential
development
of
anti-drug
antibodies
can
occur.
Velaglucerase
alfa
has
been
approved
in
several
regions,
with
brand
name
Vpriv,
and
is
one
of
several
enzyme
replacement
therapies
available
for
Gaucher
disease.
of
several
treatment
options
alongside
other
glucocerebrosidase-based
therapies.