The
FDA
approved
the
first
oral
treatment
option
for
the
autoimmune
disorder
dermatomyositis.
Brepocitinib
(Lisraya)
is
indicated
for
adults
with
the
rare
condition,
which
causes
chronic
inflammation,
progressive
muscle
weakness,
and
skin
rashes.
The
oral
drug
addresses
a
longstanding
need
for
patients
who
have
few
treatment
options
for
symptom
control.
“For
too
long,
patients
with
dermatomyositis
have
faced
a
significant
unmet
need
for
effective
treatments,
often
relying
on
therapies
meant
for
other
diseases,”
said
Nikolay
Nikolov,
MD,
of
the
FDA’s
Center
for
Drug
Evaluation
and
Research,
in
the
agency’s
press
release.
“Today’s
approval
is
a
meaningful
step
forward,
giving
patients
and
their
healthcare
providers
an
approved
oral
therapy
proven
to
help
manage
this
rare
and
debilitating
disease.”
A
TYK2/JAK1
inhibitor,
brepocitinib
helps
reduce
inflammation
known
to
damage
skin
and
muscle
tissue.
“Dermatomyositis
affects
nearly
every
aspect
of
a
patient’s
life,
causing
physical
disability,
disfiguring
skin
disease,
pain,
itch,
and
a
profound
loss
of
independence
and
sense
of
self,”
said
Ruth
Ann
Vleugels,
MD,
MPH,
of
Mass
General
Brigham
and
Harvard
Medical
School
in
Boston,
in
an
announcement
from
drugmaker
Priovant
Therapeutics.
“For
many
decades,
the
treatment
of
dermatomyositis
has
relied
on
chronic
steroids,
non-specific
immunomodulators,
and
intravenous
immunoglobulin
—
therapies
not
targeted
to
the
underlying
disease
pathobiology.
The
approval
of
Lisraya
marks
a
turning
point
for
patients
living
with
dermatomyositis.”
Support
for
the
approval
came
from
the
phase
III,
placebo-controlled
VALOR
trial
involving
241
patients
with
longstanding,
treatment-resistant
dermatomyositis.
The
trial
met
the
primary
endpoint
of
Total
Improvement
Score
(TIS)
at
52
weeks.
TIS
is
a
standardized
clinical
scoring
tool
that
tracks
changes
across
six
disease-related
domains
to
assess
a
patient’s
overall
improvement.
The
primary
analysis
showed
a
mean
TIS
of
31.2
for
placebo-treated
patients,
37.5
for
the
lower
of
two
doses
of
brepocitinib,
and
46.5
for
the
higher
dose
of
the
TYK2/JAK1
inhibitor
(P<0.001).
The
data
showed
that
45%
of
patients
treated
with
brepocitinib
discontinued
corticosteroids,
and
62%
discontinued
or
tapered
to
a
minimal
dose,
as
compared
with
38%
and
29%
of
placebo-treated
patients.
Brepocitinib
was
also
associated
with
improvement
in
multiple
patient-reported
outcomes.
The
incidence
of
adverse
events
(AEs)
was
similar
across
treatment
groups
(86-91%).
The
most
common
any-grade
AEs
in
any
group
(≥5%)
were
upper
respiratory
tract
infection,
COVID,
urinary
tract
infection,
nausea,
diarrhea,
and
headache.
Brepocitinib
will
be
available
immediately
in
the
U.S.,
according
to
the
company
announcement.
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