The
federal
340B
program
has
become
a
thorn
in
the
side
of
pharmaceutical
companies,
who
point
to
the
drug
discount
program’s
sprawling
growth
amid
what
they
contend
is
lack
of
oversight.
After
initially
proposing
changes
to
the
way
the
company
offers
drugs
under
this
program,
Johnson
&
Johnson
has
backed
down,
averting
further
confrontation
with
federal
officials
and
hospitals
on
the
matter.
For
now.
When
the
340B
program
was
established
in
1992,
the
intent
was
to
help
underserved
communities.
Under
the
program,
eligible
hospitals
and
clinics
serving
low-income
and
uninsured
patients
may
purchase
outpatient
prescription
medications
at
discounts
of
up
to
50%.
In
August,
J&J
proposed
a
major
change.
Starting
on
Oct.
15,
entities
covered
under
340B
would
have
to
pay
full
price,
then
make
a
claims
submission
for
a
rebate.
This
proposal
would
apply
to
two
drugs,
the
plaque
psoriasis
medication
Stelara
and
the
blood
thinner
Xarelto.
The
Health
Resources
and
Services
Administration
(HRSA),
which
administers
the
340B
program,
told
J&J
the
proposed
changes
are
“inconsistent”
with
the
statute
and
would
need
approval
before
they
could
take
effect.
In
a
Sept.
27
letter
to
J&J,
HRSA
Administrator
Carole
Johnson
noted
that
the
company
had
not
requested
such
approval
and
proceeding
with
the
rebate
change
would
violate
the
340B
statute.
The
administrator
added
that
implementations
of
the
rebates
would
lead
to
termination
of
the
company’s
pharmaceutical
pricing
agreement
and
a
referral
to
the
Department
Health
and
Human
Services’
Office
of
the
Inspector
General.
In
a
letter
of
response,
J&J
maintained
that
the
statute
allows
for
rebates
as
a
mechanism
for
drug
manufacturers
to
offer
the
340B
price
to
covered
entities.
Furthermore,
the
company
said
its
rebate
proposal
will
target
duplicate
discounts,
transactions
in
which
a
covered
entity
receives
a
340B
discount
and
then
submits
a
claim
for
a
Medicaid
rebate
—
a
violation
of
the
statute.
J&J
says
audits
to
uncover
such
duplicates
have
not
solved
the
problem.
“As
HRSA
knows,
J&J’s
efforts
to
identify
and
address
program
abuse
through
HRSA-approved
audits
of
covered
entities
have
been
thwarted;
nearly
all
of
the
entities
for
whom
HRSA
approved
J&J
audit
requests
have
violated
federal
law
by
refusing
to
cooperate,”
the
company’s
letter
said.
“Some
have
gone
so
far
as
to
file
suit
against
HRSA
to
invalidate
the
audit
approvals.
Plainly,
audits
alone
are
not
a
viable
means
for
J&J
to
obtain
information
necessary
to
detect
unlawful
duplicate
discounts
and
diversion
as
they
occur.”
Nevertheless,
J&J
said
HRSA’s
threat
to
terminate
the
company’s
participation
in
the
program
would
have
the
effect
of
cutting
off
millions
of
Medicare
and
Medicaid
patients
from
necessary
medicines.
The
company’s
commitment
to
these
patients
leaves
it
“no
choice
but
to
forego
implementation
of
the
rebate
model
pending
resolution
of
these
issues.”
J&J
hints
that
litigation
could
be
a
next
step.
The
company
says
it
continues
to
believe
its
rebate
model
is
“legally
permissible”
and
“sorely
needed”
for
drugmakers
to
comply
with
requirements
of
both
the
Inflation
Reduction
Act
and
the
340B
statute.
“J&J
reserves
all
of
its
legal
rights
with
respect
to
this
matter,”
the
company
said.
In
other
regulatory
news,
we
have
FDA
decisions
for
drugs
(mostly
approvals),
some
clinical
holds,
and
one
product
withdrawal.
Here’s
a
recap
of
recent
regulatory
developments:
Regulatory
Decisions
—Exact
Sciences’
Cologuard
Plus
now
has
FDA
approval.
The
product
is
a
next-generation
version
of
Cologuard,
the
company’s
non-invasive
colorectal
cancer
test.
Cologuard
Plus
offers
greater
sensitivity
for
colorectal
cancer
and
advanced
precancerous
lesions.
Like
Cologuard,
Cologuard
Plus
is
approved
for
use
in
adults
age
45
and
older
who
are
at
average
risk
of
colorectal
cancer.
Exact
Sciences
said
it
expects
to
launch
this
new
test
in
2025.
—Partners
Fresenius
Kabi
and
Formycon
received
FDA
approval
for
Otulfi,
a
biosimilar
referencing
the
Johnson
&
Johnson
biologic
drug
Stelara.
The
regulatory
decision
for
Otulfi
covers
all
of
the
approved
uses
of
Stelara,
which
include
Crohn’s
disease,
ulcerative
colitis,
moderate-to-severe
plaque
psoriasis,
and
active
psoriatic
arthritis.
Separately,
the
European
Union
approved
Otulfi
in
late
September.
Otulfi
will
jostle
for
market
share
with
Pyzchiva
from
Samsung
Bioepis
and
Sandoz,
and
Wezlana
from
Amgen,
two
Stelara
biosimilars
that
have
received
FDA
approvals
in
the
past
year.
—Eli
Lilly
drug
Retevmo
is
now
FDA
approved
for
treating
adults
and
pediatric
patients
age
2
and
older
who
have
advanced
cases
of
medullary
thyroid
cancer
carrying
a
RET
mutation.
The
drug
has
been
available
in
this
indication
under
accelerated
approval.
The
latest
regulatory
decision
converts
Retevmo’s
status
to
traditional
approval.
—Dupixent,
a
blockbuster
immunology
drug
from
Sanofi
and
Regeneron
Pharmaceuticals,
expanded
its
approved
uses
to
include
chronic
obstructive
pulmonary
disease
(COPD).
Dupixent
is
the
first
FDA-approved
biological
treatment
for
COPD.
Dupixent
approved
uses
now
span
six
dermatological
and
respiratory
conditions.
—FDA
approval
of
Bristol
Myers
Squibb
drug
Cobenfy
makes
the
drug
the
first
novel
schizophrenia
drug
in
decades.
The
twice-daily
capsule
is
designed
to
target
a
different
receptor
than
currently
available
antipsychotic
drugs,
offering
better
efficacy
and
tolerability.
Cobenfy
comes
from
Karuna
Therapeutics,
which
BMS
acquired
for
$14
billion.
—Eli
Lilly
drug
Kisunla
is
now
approved
in
Japan
for
the
treatment
of
patients
in
the
early
stages
of
Alzheimer’s
disease.
Kisunla
is
part
of
a
class
of
antibody
drugs
that
work
by
reducing
plaques
of
amyloid
proteins
that
form
in
the
brains
of
Alzheimer’s
patients.
Japan
marks
the
second
major
market
approval
for
Kisunla,
which
was
approved
by
the
FDA
in
July.
—In
other
Japanese
regulatory
news,
Takeda
Pharmaceutical’s
Fruzaqla
won
approval
for
the
treatment
of
advanced
or
recurrent
colorectal
cancer.
The
Japanese
pharma
giant
acquired
the
oral
small
molecule
from
Hutchmed
in
early
2023.
Nearly
a
year
ago,
the
FDA
approved
the
drug
for
treating
colorectal
cancer.
—Ipsen
landed
European
Union
approval
for
Kayfanda
as
a
treatment
for
cholestatic
pruritus
(severe
itching)
associated
with
the
rare
liver
disease
Alagille
syndrome.
The
approval
was
made
under
“exceptional
circumstances”
that
permits
marketing
authorization
when
comprehensive
efficacy
and
safety
data
are
not
available.
The
Ipsen
drug
won
FDA
approval
for
Alagille
patients
last
year;
in
the
U.S.,
the
once-daily
capsule
is
marketed
as
Bylvay.
Ipsen
added
the
drug
to
its
pipeline
via
its
$952
million
acquisition
of
Albireo.
—Ipsen’s
rare
liver
disease
portfolio
also
welcomed
European
Union
approval
of
Iqirvo
for
treating
primary
biliary
cholangitis
(PBC),
a
chronic
disorder
that
damages
the
liver’s
bile
ducts.
The
approval
covers
use
of
the
once-daily
pill
in
combination
with
ursodeoxycholic
acid
(UDCA)
for
patients
whose
disease
does
not
adequately
respond
to
that
standard
of
care
drug.
Iqirvo
may
be
used
as
a
monotherapy
for
patients
who
cannot
tolerate
UDCA.
European
approval
comes
nearly
four
months
after
the
FDA
greenlit
Iqirvo
for
PBC.
—Zevra
Therapeutics
drug
Miplyffa
became
the
first
FDA-approved
treatment
for
Niemann-Pick
disease
type
C,
a
rare
inherited
lysosomal
storage
disorder
that
can
become
fatal
by
the
time
patients
reach
adolescence.
The
approval
specifically
covers
the
neurological
effects
of
the
disease.
Zevra
acquired
the
rights
to
Miplyffa
from
Orphazyme,
which
failed
to
win
FDA
approval
for
the
small
molecule
in
2021.
—Days
after
Miplyffa’s
approval,
the
FDA
approved
levacetylleucine,
brand
name
Aqneursa,
making
the
drug
from
privately
held
IntraBio
the
second
approved
treatment
for
Niemann-Pick
disease
type
C.
Similar
to
Miplyffa,
the
regulatory
decision
for
the
IntraBio
drug
covers
the
treatment
of
neurological
effects
of
the
rare
disease.
—The
European
Union
approved
Astellas
drug
zolbetuximab,
brand
name
Vyloy,
as
a
treatment
for
advanced
gastric
and
gastroesophageal
junction
cancer.
The
approval
covers
use
of
the
drug
in
combination
with
chemotherapy.
The
FDA
turned
down
Astellas’s
submission
for
the
drug
early
this
year,
citing
manufacturing
issues.
Astellas
resubmitted
its
application,
which
now
has
a
Nov.
9
target
date
for
a
regulatory
decision.
—FluMist,
an
intranasally
dosed
influenza
vaccine
that’s
been
available
in
the
U.S.
for
two
decades,
is
now
FDA-approved
for
self-
or
caregiver
administration.
The
regulatory
decision
makes
the
AstraZeneca
product
the
first
influenza
vaccine
that
does
not
need
to
be
given
by
a
healthcare
provider.
For
those
choosing
this
option,
the
vaccine
will
be
available
through
a
third-party
pharmacy
following
a
screening
and
eligibility
assessment
completed
when
patients
order
the
vaccine.
—The
FDA
rejected
Vanda
Pharmaceuticals’
tradipitant
as
a
treatment
of
gastroparesis.
This
delayed
gastric
emptying
is
associated
with
diabetes
but
can
also
develop
in
those
who
do
not
have
diabetes.
According
to
Vanda,
the
FDA
disregarded
clinical
evidence
for
the
drug
and
asked
the
company
to
conduct
additional
clinical
testing.
The
company
said
it
still
plans
to
seek
marketing
authorization
for
this
drug.
A
separate
new
drug
application
is
also
planned
for
tradipitant
later
this
year
for
preventing
vomiting
from
motion
sickness.
—An
experimental
Applied
Therapeutics
drug
for
the
rare
genetic
metabolic
disease
classic
galactosemia
will
not
be
discussed
by
an
FDA
advisory
committee.
After
completing
a
late-cycle
review
meeting,
the
agency
told
the
company
a
meeting
was
no
longer
needed
for
the
drug,
govorestat.
The
Nov.
28
target
date
for
a
regulatory
decision
still
stands.
—The
FDA
authorized
the
first
over-the-counter
hearing
aid
software.
Called
Hearing
Aid
Feature,
this
Apple
software
is
compatible
with
the
company’s
AirPods
Pro
headphones.
The
software
was
reviewed
through
the
regulator’s
De
Novo
premarket
review
pathway.
FDA
authorization
is
based
on
a
clinical
trial
enrolling
118
patients
with
mild-to-moderate
hearing
loss.
Results
showed
those
who
self-fit
the
software
and
device
achieved
similar
benefit
as
those
who
received
a
professional
fitting.
—Eli
Lilly
won
FDA
approval
for
lebrikizumab,
brand
name
Ebglyss,
a
new
treatment
for
atopic
dermatitis.
It
will
compete
with
biologic
drugs
Dupixent
from
Sanofi
and
Regeneron
Pharmaceuticals,
and
Adbry
from
LEO
Pharma.
Compared
to
those
every-other-week
injectable
drugs,
Lilly’s
new
product
offers
patients
less
burdensome
once-monthly
maintenance
dosing.
—Roche
landed
FDA
approvals
for
subcutaneously
injectable
drugs
that
were
originally
developed
as
intravenous
infusions.
Tecentriq
Hybreza
is
the
injectable
version
of
the
cancer
immunotherapy
Tecentriq;
Ocrevus
Zunovo
is
the
injectable
version
of
the
multiple
sclerosis
drug
Ocrevus.
Both
drugs
employ
drug
delivery
technology
from
Halozyme
that
enables
biologic
drugs
to
be
administered
as
injections.
—Travere
Therapeutics
drug
Filspari
converted
its
accelerated
approval
into
a
full
FDA
approval
for
the
treatment
of
the
rare
kidney
disease
immunoglobulin
A
nephropathy.
The
regulatory
decision
came
nearly
a
year
after
Travere
reported
the
drug
narrowly
failed
its
Phase
3
confirmatory
study.
The
approval
is
based
on
longer-term
data
showing
the
once-daily
pill
significantly
slowed
the
kidney
function
decline.
One
Clinical
Hold
Placed,
Two
Lifted
—Following
the
report
of
patient
deaths,
the
FDA
formally
placed
a
clinical
hold
on
zetomipzomib,
a
Kezar
Life
Sciences
drug
in
development
for
treating
lupus
nephritis.
Kezar
had
voluntarily
stopped
dosing
and
enrollment
in
the
study
after
emerging
safety
data
showed
four
Grade
5
(fatal)
serious
adverse
events
over
the
course
of
the
study
so
far.
The
company
said
that
three
of
those
fatalities
showed
a
common
pattern
of
symptoms
and
proximity
to
dosing;
additional
non-fatal
complications
also
showed
a
proximity
to
dosing.
—The
FDA
lifted
a
clinical
hold
on
Biomea
Fusion’s
Phase
1/2
clinical
trial
testing
BMF-219
in
type
1
and
type
2
diabetes.
Redwood
City,
California-based
Biomea
said
review
of
the
clinical
data
to
date
found
that
concerning
safety
signals
observed
in
the
Phase
2a
escalation
study
did
not
translate
to
the
larger
Phase
2b
expansion
study.
BMF-219
is
a
small
molecule
that
forms
a
covalent
bond
and
inhibits
menin,
a
protein
thought
to
suppress
the
pancreatic
beta
cells
that
produce
and
secrete
insulin.
—The
FDA
removed
the
partial
clinical
hold
placed
on
an
Avidity
Biosciences’
delpacibart
etedesiran,
or
del-disiran,
an
RNA
therapy
in
development
for
the
rare
muscular
disorder
myotonic
dystrophy
type
1.
The
partial
hold
was
placed
in
2022
following
the
report
of
a
serious
adverse
event
in
a
single
patient.
Del-disiran
is
currently
in
Phase
3
testing.
A
Rare
Disease
Drug
Withdrawal
—Pfizer
is
voluntarily
withdrawing
Oxbryta
from
the
market
and
discontinuing
all
clinical
tests
of
the
drug,
which
won
accelerated
FDA
approval
for
sickle
cell
disease
in
2019.
The
pharma
giant
said
its
decision
is
based
on
the
totality
of
data
now
indicating
higher
rates
of
complications
and
deaths
in
patients
treated
with
the
oral
drug.
The
FDA
issued
an
alert
about
the
withdrawal
and
said
it
has
been
conducting
its
own
safety
review
of
postmarketing
data.
When
the
review
concludes,
the
agency
said
it
will
communicate
additional
findings,
if
necessary.
Oxbryta
was
the
centerpiece
of
Pfizer’s
$5.4
billion
acquisition
of
Global
Blood
Therapeutics
in
2022.
Photo:
Mario
Tama,
Getty
Images
Kathryn
And
