FDA approves Pfizer’s first gene therapy for rare inherited bleeding disorder

The
Food
and
Drug
Administration

on
Friday
approved


Pfizer‘s
treatment
for
a
rare
genetic

bleeding
disorder,
making
it
the
company’s

first-ever
gene
therapy
to
win
clearance
in
the
U.S. 

The
agency

greenlit
the
drug,
which
will
be
marketed
as
Beqvez,
for
adults
with
moderate
to
severe

hemophilia
B
who
meet
certain

requirements.

The
treatment
will
be
available
by
prescription
to
eligible
patients
this
quarter,
a
Pfizer
spokesperson
told
CNBC.
It
has
a
hefty
$3.5
million
price
tag,
before
insurance
and
other
rebates,
the
spokesperson
added,
making
it
by
far
one
of
the
most
expensive
drugs
in
the
U.S.

More
than
7,000
people
in
the
U.S.
are
living
with
the
debilitating

condition,
which
predominantly
affects
men,
according
to
an

advocacy
group.
The
condition
is
caused
by
insufficient
levels
of
a

certain
protein
that
helps
blood
form
clots
to
stop
bleeding
and
seal
wounds.
Without
that
protein,
called
factor
IX,
patients
with
hemophilia
B
bruise
easily
and
bleed
more
frequently
and
for
longer
periods
of
time. 

Beqvez
is
a
one-time
treatment
designed
to
enable
patients
to
produce
factor
IX
themselves
and
prevent
and
control
bleeding. In
a
late-stage
trial,
the
drug
was
superior
to
the
often-cumbersome

standard
treatment
for
hemophilia
B,
which
involves
administering
the
protein
multiple
times
a
week
or
a
month
through
the
veins. 

“Many
people
with
hemophilia
B
struggle
with
the
commitment
and
lifestyle
disruption
of
regular
[factor
IX]
infusions,
as
well
as
spontaneous
bleeding
episodes,
which
can
lead
to
painful
joint
damage
and
mobility
issues,”
said
Dr.
Adam
Cuker,
director
of
Penn’s
Comprehensive
and
Hemophilia
Thrombosis
Program,
in
a
Pfizer

release
on
Friday.

Pfizer’s
drug “has
the
potential
to
be
transformative
for
appropriate
patients
by
reducing
both
the
medical
and
treatment
burden
over
the
long
term,”
Cuker
added. 

The
approval
is
a
big
step
for
Pfizer,
which
is
trying
to
regain
its
footing
following
the
rapid
decline
of
its
Covid
business
last
year.
The
company
is

betting
big
on
cancer
drugs
and
treatments
for
other
disease
areas
to
help
turn
its
business
around. 

Pfizer
is
one
of
several
companies
to
invest
in
the
rapidly
growing
field
of
gene
and
cell
therapies.
They
are
one-time,
high-cost
treatments
that
target
a
patient’s
genetic
source
or
cell
to
cure
or
significantly
alter
the
course
of
a
disease.
Some
health
experts
expect
cell
and
gene
therapies
to
replace
traditional
lifelong
treatments
that
people
take
to
manage
chronic
diseases. 

Pfizer

gained
the
rights
to
produce
and
market
Beqvez
from
Spark
Therapeutics
in
2014. 

The
company
is
offering
payers
a
warranty
program
to
cover
patients
who
receive
Beqvez,
a
spokesperson
told
CNBC.
Pfizer
expects
that
program
to
offer “financial
protections
by
insuring
against
the
risk
of
efficacy
failure,”
they
added.

The
gene
therapy
will
compete
with
Australia-based
CSL
Behring’s

Hemgenix,
a
similar
treatment
that

won
FDA
approval
for
hemophilia
B
in
2022.
That
drug
has
a
similar
list
price
of
$3.5
million
in
the
U.S.,
before
insurance
and
other
rebates. 

Notably,
some
health
experts
have
said
that
high
costs
and
logistical
issues,
among
other
factors,
have

limited
the
uptake
of
Hemgenix
and
another
approved
gene
therapy
for
the
more
common
hemophilia
A. 

Pfizer
also
seeks
FDA
approval
for
its
experimental
antibody,
marstacimab,
to
treat
hemophilia
A
and
B.
The
company
is
also
developing
a
gene
therapy
for
Duchenne
muscular
dystrophy,
a
genetic
disorder
that
causes
muscles
to
weaken
gradually.