The
FDA
approved
nipocalimab
(Imaavy)
as
the
first
treatment
for
warm
autoimmune
hemolytic
anemia
(wAIHA),
the
most
common
form
of
autoimmune
hemolytic
anemia.
Approval
of
the
immunoselective
neonatal
Fc
receptor
(FcRn)
blocker
stipulates
use
in
adult
and
pediatric
patients
ages
12
years
and
up
who
have
received
corticosteroids
or
were
previously
treated
with
them.
Until
now,
standard
treatments
for
wAIHA
have
included
corticosteroids
and
immunosuppressants,
which
don’t
target
the
underlying
cause
of
the
disease
—
the
immunoglobulin
G
(IgG)
autoantibodies
that
flag
red
blood
cells
for
destruction
by
the
immune
system.
Severe
anemia
and
its
sequelae
follow,
increasing
the
risks
for
morbidity
and
mortality.
Nipocalimab
reduces
pathogenic
IgG
autoantibodies
but
preserves
B-cell
function.
Unlike
in
cold
agglutinin
disease
—
another
rare
hemolytic
anemia
—
hemolysis
in
wAIHA
occurs
at
normal
temperatures
or
above,
hence
the
“warm”
moniker.
The
approval
was
supported
by
ENERGY,
a
placebo-controlled
phase
II/III
trial
that
enrolled
wAIHA
patients
with
low
hemoglobin
(Hgb)
levels
(<10
g/dL),
evidence
of
hemolysis,
and
a
positive
direct
antiglobulin
test.
Patients
could
stay
on
background
medications
for
wAIHA.
At
24
weeks,
a
greater
number
of
patients
in
the
30
mg/kg
nipocalimab
arm
(delivered
intravenously
every
4
weeks)
achieved
a
durable
Hgb
response
versus
the
placebo
arm
(24%
vs
8%,
respectively),
with
responses
defined
as
an
Hgb
concentration
of
at
least
10
g/dL
plus
an
increase
of
2
g/dL
or
more
from
baseline
for
at
least
28
days.
Some
improvements
in
fatigue
were
also
associated
with
nipocalimab
treatment.
“The
phase
II/III
ENERGY
study
demonstrates
that
targeting
pathogenic
IgG
can
meaningfully
change
the
treatment
paradigm
for
wAIHA,”
said
David
Kuter,
MD,
DPhil,
of
Harvard
Medical
School
in
Boston,
in
a
statement
from
drugmaker
Johnson
&
Johnson.
“For
patients,
this
approval
means
that
they
now
have
a
therapy
with
a
proven
safety
profile
that
targets
the
disease-driving
autoantibodies
in
wAIHA.”
According
to
the
labeling,
common
adverse
events
in
wAIHA
trials
of
nipocalimab
included
peripheral
edema,
diarrhea,
and
pyrexia
—
each
occurring
in
at
least
10%
of
patients.
The
warnings
and
precautions
section
also
notes
potential
risks
for
infections,
hypersensitivity
reactions,
and
infusion-related
reactions
such
as
influenza-like
illness,
chills,
or
nausea.
Nipocalimab
was
first
approved
last
year
for
generalized
myasthenia
gravis.
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