FDA approves Merck’s drug for rare, deadly lung condition

The
Food
and
Drug
Administration
on
Tuesday

approved
a
drug
from


Merck
designed
to
treat
a
progressive
and
life-threatening

lung
condition
in
a
win
for
both
the
drugmaker
and
for
patients
suffering
from
the
rare
disease.

The
agency
greenlighted
the
therapy,
which
will
be
marketed
as
Winrevair,
for
adults
with

pulmonary
arterial
hypertension.
The
decision
is
a
big
step
for
the
roughly

40,000
people
in
the
U.S.
living
with
that
disease
because
Winrevair
is
the
first
drug
to
target
the
root
cause
of
the
condition. Other

available
medicines
only
help
manage
symptoms. 

The
condition
refers
to
when
the
small
blood
vessels
in
the
lungs
narrow.
That
leads
to
high
blood
pressure
in
the
arteries
that
carry
blood
from
the
heart
to
the
lungs,
which
can
damage
the
heart
and
result
in
limited
physical
activity.
Starting
from
diagnosis,
the
mortality
rate
of
patients
is
43%
by
five
years,
according
to
Merck.

Merck
estimates
that
Winrevair
will
be
available
in
select
specialty
pharmacies
in
the
U.S.
by
the
end
of
April,
according
to
a
company
release.
The
drug
is
an
injection
administered
every
three
weeks
and
is
distributed
in
single-vial
or
double-vial
kits.

It
will
priced
at
$14,000
per
vial
before
insurance,
a
Merck
spokesperson
said
in
a
statement.
But
the
company
has
a
program
that
offers
eligible
patients
help
with
out-of-pocket
costs
and
copays.

Winrevair
is
meant
to
be
used
along
with
existing
therapies
for
the
condition
to
increase
exercise
capacity,
lessen
the
severity
of
PAH
and
reduce
the
risk
of
the
disease
worsening.

The
approval
is
critical
for
Merck,
which
is
working
to
diversify
its
revenue
stream
as
its
top-selling
cancer
immunotherapy

Keytruda
approaches
a
loss
of
market
exclusivity
in
2028. 

In
a
note
this
month,
JPMorgan
analyst
Chris
Schott
estimated
that
Winrevair
would
reach
worldwide
annual
sales
of
around
$5
billion
by
2030
and
emerge
as
one
of
Merck’s “largest
growth
drivers.” 

Merck
Chief
Medical
Officer
Eliav
Barr
told
CNBC
that “this
is
a
really
great
opportunity
for
the
company,
but
really,
more
importantly,
a
great
important
opportunity
for
patients.”
He
noted
that
the
drug
will
be
a “paradigm
shift”
for
patients
living
with
PAH.

The
company
gained
the
rights
to
Winrevair
through
its

$11.5
billion
acquisition
of
Acceleron
Pharma
in
2021.
At
the
time,
Merck
estimated
that
PAH
would
be
a
roughly
$7.5
billion
market
by
2026. 

The
FDA’s
approval
is
based
on
data
from
a

late-stage
trial,
which
followed
more
than
300
patients
at
a
moderate
stage
of
PAH
who
were
already
taking
another
medication
for
the
blood
vessel
condition. 

The
study
found
that
Winrevair
combined
with
an
existing
therapy
helped
patients
with
the
condition
walk
about
40.8
meters
more
in
six
minutes
than
those
who
received
a
placebo,
24
weeks
into
the
trial. 

“There
is
tremendous
improvement
in
people’s
ability
to
exercise
and
move
around,”
Barr
said. “Because
this
disease
causes
people
to
be
very,
very
homebound.
They
have
shortness
of
breath,
they
can’t
move.” 

Winrevair
on
top
of
an
existing
medication
also
significantly
improved
eight
of
nine
secondary
goals
in
the
study.
That
includes
reducing
the
risk
of
death
or
worsening
of
the
condition
by
84%
compared
to
an
existing
drug
alone.

Severe
and
serious
adverse
events
were

less
common
in
the
group
of
patients
who
took
Winrevair
compared
to
those
who
received
a
placebo,
according
to
the
trial. Side
effects
that
occurred
more
frequently
included
nose
bleeds,
headaches
and
rashes,
among
others.

One
notable
advantage
of
Winrevair
is
that
patients
or
caregivers
can
inject
it
under
the
skin
with
appropriate
training
from
a
healthcare
provider.
Meanwhile,
some
existing
treatments
for
PAH
must
be
administered
by
medical
professionals
at
an
infusion
center. 

“One
of
the
things
we
heard
very
loud
and
very
clear,
from
both
patients
and
physicians,
is
that
they
wanted
something
that
you
could
get
at
home,”
Barr
said. 

Merck
is
continuing
to
study
Winrevair
in
other
phase
two
and
phase
three
trials.

Those
trials
include

late-stage
studies
on
patients
with
more
advanced
PAH
disease,
and
those
who
are
within
the
first
year
after
diagnosis.
Merck
has
said
it
expects
those
trials
to
finish
around
2025
and
2026.