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1
White paper
Jefferies 2012: Global Healthcare Conference Review
By Caroline Richards & Maura MusciaccoNovember 2012
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Datamonitor HealthcarePharma intelligence |
2
DatamGlobaLondo This analy
Prod
Lundbec
The
breast ca
(insulin d
trastuzum
rheumato
hypercho
bowel sy
84]) for t
ceftoloza
complica
infusion
monitoal Healton oveysis review co
duct-specific
ck, ImmunoG
status of late
ancer, Novo
degludec + in
mab emtans
oid arthritis, A
olesterolemia
yndrome and
he treatment
ane/tazobact
ated intra-abd
or attenthcare
er Noveovers the key
and overall
Gen, Medivat
e-stage agen
Nordisk’s Tr
nsulin aspart
ine (TDM1) f
Aegerion’s lo
a, NPS Phar
d Natpara (re
t of adult hyp
tam for comp
dominal infec
nded th Confe
ember y highlights o
company up
ion, Novo No
nts such as R
resiba (insuli
t) for type 2 d
for breast ca
opidomide fo
rmaceuticals’
ecombinant h
poparathyroid
plicated urina
ctions, and U
he Jeffeerence 14–15,of the confer
pdates for: Ae
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n degludec)
diabetes, Imm
ncer, Rigel’s
or homozygo
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ary tract infec
United Thera
eries 2held in, 2012.
rence includin
egerion, Cub
jeta (pertuzu
and Ryzode
munoGen’s
s fostamatinib
us familial
uglutide) for
hyroid hormo
t’s
ctions and
peutics’ impl
2012 n . ng:
bist,
mab) for
eg
b for
short
one [1-
lantable
Datamonitor HealthcarePharma intelligence |
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The quanswe
directly Aeg
prescreat
inte
Key prgloba
Lomitapide
homozygou
characteriz
treatments
nature of its
developme
date of Dec
adults with
that the dru
(LDL-C) an
Aegerion a
EU a MAA
company e
uestion aner sessionproceedi
gerion’s entation ted some eresting
resental healt
e is Aegerion
us familial hy
zed by extrem
. Aegerion d
s promising
ental and reg
cember 29, 2
HoFH and p
ug led to a 50
nd had a favo
also briefly dis
has been file
expects appro
nd n ing
ations thcare
’s lead produ
ypercholeste
mely high cho
edicated its 4
new candida
ulatory timel
The US
curr
A
c
A
r
to
make
drug on the
2012. Aegeri
presented 78
0.2% reducti
orable risk-be
scussed lopi
ed with the E
oval to come
Aegappdatdiso
from Jconfer
uct, and has
rolemia (HoF
olesterol and
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ate for the dis
ines.
S Food and D
rently review
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company hig
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ecommende
two vote in f
es it likely tha
e Prescription
on has comp
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on in low-de
enefit profile
idomide’s sta
European Me
e about in mi
gerion: hoproval chee for drugorder loom
Jefferierence
just complet
FH), a rare d
d with no cur
resentation to
sease and su
Drug Adminis
ing a Market
MAA) for lopid
ghlighted that
gic and Meta
mmittee (EM
ed approval o
favor of back
at the FDA w
n Drug User
pleted a pivo
from this stu
ensity lipopro
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atus in ex-US
edicines Age
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opes for neeer intensig to treat rms
es 2012
ted Phase III
disorder
rrent effective
o a round-up
ummarized e
stration (FDA
ting Authoriz
domide. The
t the FDA's
abolic Drugs
MDAC) recen
of lopidomide
king the drug
ill now appro
Fee Act (PD
otal Phase III
dy, which sh
tein choleste
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ency (EMA), a
anwhile, Aeg
ew-year ify as PDUrare chole
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e
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ove the
DUFA)
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In the
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erion is
UFA esterol
Datamonitor HealthcarePharma intelligence |
4
gearing up
year. This w
The questio
spun up so
Pharmaceu
approachin
lopidomide
factor betw
oral drug w
drug and so
to start Phas
will be a sma
on and answ
ome interestin
uticals, whos
ng approval.
e, at the end o
ween its HoFH
with a half-life
o carries the
se I trials in J
all study, in fe
wer session d
ng discussio
se late-stage
Mipomersen
of January 2
H product an
e of 20–30 ho
e risk of inject
Japanese pa
ewer than 10
directly proce
n on Aegerio
HoFH cand
n’s PDUFA d
2013. Aegerio
nd ISIS’s is th
ours, wherea
tion-site reac
atients towar
0 patients.
eeding Aeger
on’s main co
idate, mipom
ate is a little
on said that a
hat lopidomid
as Mipomers
ctions.
rds the end o
rion’s presen
mpetitor, ISI
mersen, is als
later than th
a differentiat
de is a once-
en is an inje
of this
ntation
S
so
hat of
ting
-daily
ctable
Datamonitor HealthcarePharma intelligence |
5
Cubist star
time it hope
late-stage t
income. It i
three cand
treatment f
receptor an
and its intra
(CXA-201)
urinary and
While it is t
in 5 years'
revenues w
while non-
to $77.7m,
The compa
product Cu
drug was a
treatment o
infections,
Staphyloco
usually occ
product in a
Cubist attri
rted its prese
es to have ge
trials, and to
s already pa
idates in Pha
for Clostridiu
ntagonist, CB
avenously ad
for the treat
d reproductiv
too early to s
time, it certa
were up 18%
GAAP adjus
compared to
any turned ne
ubicin (daptom
approved in t
of complicate
including me
occus aureus
cur in hospita
a new class
buted its stro
Cubunvdeli
entation by ou
enerated $2b
have achiev
art of the way
ase III develo
m difficile-as
B-5945, for th
dministered c
ment of com
ve tract infect
surmise on w
ainly posted s
% in Q3 2012
sted operatin
o $71.2m in
ext to discus
mycin for inje
he US in 200
ed skin and s
ethicillin-resis
s (MRSA). Th
alized patient
of antibiotics
ong Q3 2012
bist: ambiveiled as Civer reven
utlining its vis
bn in global r
ved $700m in
y there with it
opment: an o
ssociated dia
he treatment
combination
mplicated intra
tions.
whether Cubis
solid Q3 201
over the sam
g income inc
Q3 2011.
ss its approve
ection). This
03 for the
skin structure
stant
hese serious
ts and Cubic
s called cyclic
2 revenue gro
itious 5-yeCubicin conue growt
g
sion for the f
revenue, hav
n non-GAAP
ts pipeline as
oral lipopepti
arrhea (CDAD
t of opioid-ind
of ceftolozan
a-abdominal
st will achiev
2 financial re
me period in
creased 9%
ed
e
s infections
cin was the fi
c lipopeptide
owth
ear plan ontinues tth
In 5 yea[Cubist]
to hgenerate
in glo
reve
future: in 5 ye
ve four candi
adjusted op
spirations. It
de, CB-315,
D); a mu-opio
duced consti
ne and tazob
infections an
ve its financia
esults. Its tot
2011, at $23
rst approved
e antibiotic ag
to
ars' time ] hopes
have ed $2bn obal nue
ears'
idates in
perating
has
for the
oid
ipation;
bactam
nd
al goals
al net
38.2m,
d
gents.
Datamonitor HealthcarePharma intelligence |
6
Cubist fina
ceftolozane
infections a
product has
against a n
particular b
explained,
currently av
infections.
qualified in
Antibiotic In
from the FD
designation
Hatch-Wax
lized its pres
e/tazobactam
and complica
s the potenti
number of ba
bacterium is e
and has dev
vailable antib
In October 2
fectious dise
ncentives No
DA is expect
ns would qua
xman exclusi
sentation by d
m, which is in
ated intra-abd
al to be more
acterial strain
extremely pr
veloped num
biotics, makin
2012, Cubist
ease product
ow (GAIN) ac
ted within 60
alify CXA-20
ivity, priority
discussing it
n Phase III tr
dominal infec
e successful
ns, including
revalent in th
erous resista
ng it one of p
submitted ap
t (QIDP) des
ct for both CX
days of sub
1 and CB-31
review, and
s combinatio
ials for comp
ctions. Cubis
than Cubici
Pseudomona
e hospital se
ance mechan
physicians’ m
pplications to
ignation und
XA-201 and
mission. If g
5 for a 5-yea
eligibility for
on drug
plicated urina
st stated that
n, since it is
as aeruginos
etting, Cubist
nisms agains
most feared
o the FDA fo
er the Gener
CB-315. A re
ranted, the
ar extension
fast-track sta
ary tract
t this
active
sa. This
t
st
r
rating
esponse
of
atus.
Datamonitor HealthcarePharma intelligence |
7
PerjetT-DM
expecteparticprom
Roche’s CF
illustrating
solid growt
company in
has a subs
are in Phas
three cand
approved in
of Europe;
Europe; an
Europe. Pe
enable Roc
franchise. P
potential to
metastatic
antibody-dr
activity whi
(see Immu
sales grew
experience
Europe wa
impact of g
Bonviva (ib
ta and M1 are ed to be cularly
mising
FO, Alan Hip
a considerab
th in the eme
nvests nearly
stantial pipeli
se I, 24 are in
idates are un
n the US and
Erivedge (vi
nd T-DM1 (tra
erjeta and T-
che to build o
Perjeta in co
o create a ne
breast cance
rug conjugat
le targeting i
noGen below
L
Centra
w by 12% in b
ed only single
s the only re
generic erosio
bandronic ac
ppe, presente
ble pipeline a
erging marke
y half of this
ne with 71 n
n Phase II, a
ndergoing re
d Switzerland
smodegib), w
astuzumab e
DM1 are exp
on its human
mbination w
w standard o
er. T-DM1, w
tes, or “arme
intracellular d
w).
Year-to-da
generated
company’
Asia grew
Latin Americ
al Asia, Midd
both regions.
e-digit growth
egion to unde
on of produc
id).
F. Hoffmaassets to
ed an overvie
and strong fo
ts. With a tot
into its late-s
ew molecula
and eight are
gistration: Pe
d and is curr
which is app
emtansine), w
pected to be
n epidermal g
ith Herceptin
of care for wo
which is an e
d antibodies
delivery of a
ate sales illus
d in the emer
’s pharmaceu
by 14% (driv
ca and CEMA
le East, Afric
In stark con
h (6% and 1%
ergo a contra
cts such as C
an La Rocbuild on
ew of the Ro
ocus on onco
tal R&D budg
stage assets
ar entities (NM
e in Phase III
erjeta (pertuz
rently being r
roved in the
which is filed
particularly p
growth factor
n (trastuzuma
omen with H
merging clas
s” – retains H
potent cell-k
strate the stro
rging market
uticals divisio
ven mostly b
AI (Central an
ca, and India
trast, sales i
%, respective
action in sale
CellCept (myc
che: late-sits Hercep
oche Group,
ology, as wel
get of CHF8
. Currently R
MEs), of whi
. The remain
zumab), whi
reviewed in t
US and filed
in the US an
promising as
r receptor 2 (
ab; Roche) h
HER2-posiitve
ss of therapie
Herceptin’s bi
killing agent,
ongest growt
ts. Looking a
on only, sale
by China), wh
nd Eastern E
an subcontine
n the US and
ely), while W
es (-2%) due
cophenolate
tage pipeptin franc
ll as
bn, the
Roche
ch 36
ning
ch is
he rest
d in
nd in
s they
(HER2)
has the
e
es –
iological
DM1
th being
at the
es in
hile in
Europe,
ent)
d Japan
Western
to the
) and
line hise
Datamonitor HealthcarePharma intelligence |
8
Lundbeck i
products in
portfolio an
substantial
major oppo
Revenue fr
Sabril (viga
episodes a
in Japan, O
lymphocytic
revenues. I
comparing
in sales in t
launched in
Lundbeck’s
than 50% o
by three lat
launch duri
(aripiprazo
(nalmefene
antidepress
US, Europe
2013. The
registration
2013, while
addition the
bipolar diso
of alcohol c
feedback fr
expected in
s shifting aw
n the Europea
nd a balance
unrealized p
ortunity for Lu
rom new pro
abatrin; epile
associated wi
Onfi (clobaza
c leukemia) d
Its key brand
Q3 2011 sa
this market d
n Japan in 20
s new produc
of revenues b
te-stage pipe
ing 2013: vo
le; schizophr
e; alcohol de
sant vortioxe
e, and Canad
antipsychotic
n with the FD
e in Europe s
e drug is und
order. Selinc
consumption
rom the Com
n Q4 2012, w
way from its o
an markets,
d geographic
potential outs
undbeck, and
ducts – Xena
psy], Sycres
ith bipolar I d
am; seizures]
doubled in Q
d Lexapro ha
les to Q3 20
due to gener
011, where it
cts are expec
by 2015. Thi
eline assets t
rtioxetine (de
renia) once m
pendency). I
etine was sub
da, and Lund
c treatment A
DA in Septem
submission o
dergoing Pha
cro is expecte
n; an MAA wa
mmittee for M
with approval
H. LuLexap
old business
and instead
c footprint, g
side of Europ
d it will conti
azine (tetrab
st (asenapine
disorder], Lex
], and Treand
Q3 2012 and
as lost patent
12, this bran
ic erosion. N
t is still unde
cted to contr
s will be driv
that are expe
epression), A
monthly, and
In 2012 the
bmitted for re
dbeck is exp
Abilify once m
mber 2012, w
of an MAA is
ase III trials f
ed to be the f
as submitted
Medicinal Pro
l in H1 2013.
undbeck: spro expiry
model focus
building a m
iven that the
pe. As such,
nue its expa
benazine; Hu
e; moderate t
xapro (escita
da (bendamu
now represe
t protection i
nd experience
Nevertheless,
ergoing solid
ribute more
ven in part
ected to
Abilify
d Selincro
egistration in
ecting appro
monthly was
with approval
expected by
for acute sch
first treatmen
d in Decembe
ducts for Hu
.
shifting foy
Lundproexp
contrtha
reven
sed on a han
more diversifie
e company ha
China repre
nsion in this
ntington’s dis
to severe ma
alopram; dep
ustine; chron
ent 17% of co
n the US, an
ed an 89% d
, Lexapro wa
uptake.
the
oval by the en
submitted fo
expected du
y the end of 2
izophrenia a
nt to target re
er 2011, and
man Use (C
ocus after
dbeck’s newoducts are pected to ribute moran 50% of
nues by 201
ndful of
ed
as
esents a
market.
sease],
anic
pression]
nic
ompany
nd by
decline
as
nd of
or
uring H1
2012. In
and
eduction
HMP) is
w
re
15
Datamonitor HealthcarePharma intelligence |
9
Its leadT-DM1
regismetas
positiveand Im
expeapprov
ImmunoGe
proprietary
licenses its
antibodies;
Healthcare
trastuzuma
attached to
DM1 is und
ImmunoGe
in H2 2013
T-DM1’s co
In addition
companies
these are:
cancer, Ph
targets fola
ding candiis undergstration fostatic HER breast ca
mmunoGeecting FDAval in H1 2
en is a leadin
y Targeted An
s technology
in fact it has
e, and Sanofi
ab emtansine
o the HER2-b
dergoing reg
en is expectin
3.
ommercial po
to several pi
s, ImmunoGe
IMGN901, w
ase II, and m
ate receptor a
idate going or R2-ancer, en is A
2013
ng player in th
ntibody Paylo
to other com
s secured ke
. ImmunoGe
e (T-DM1), w
binding antib
istration for m
ng FDA appr
In add
an
ex
antibodi
otential throu
ipeline candi
en has three
which targets
multiple myel
alfa, which is
he antibody-
oad (TAP) te
mpanies for u
ey deals with
en’s leading p
which acts as
ody, Hercep
metastatic H
roval in H1 2
dition, the co
n adjuvant th
positive bre
expected
mechanis
also show
gastric ca
are expec
successful t
pansion stra
ies, ImmunoG
ugh its strate
idates that a
wholly owne
CD56-expre
oma, Phase
s over-expres
Imne
-drug conjuga
echnology. Im
use with their
Roche, Amg
pipeline cand
s a cancer-kil
ptin (trastuzum
ER2-positive
013, followed
mpound is b
herapy for ea
east cancer,
for 2018. Th
sm of action
wn potential
ancer, and re
cted in 2015.
track record
ategies for its
Gen should b
gic partner o
re being co-d
ed compound
essing cance
I); IMGN853
ssed on the
mmunoGeears the m
ate field than
mmunoGen o
r engineered
gen, Bayer
didate is
ling agent w
mab; Roche)
e breast canc
d by EMA ap
being investig
arly-stage HE
with results
hanks to its
, the therapy
in HER2-pos
esults from th
Given Roch
of indication
s own monoc
be able to m
of choice.
developed w
ds. Specifica
ers (small cel
3 (Phase I) w
most prevale
en: first prmarket
nks to its
out-
when it is
). T-
cer, and
pproval
gated as
ER2-
y has
sitive
his trial
e’s
clonal
aximize
with other
lly,
ll lung
which
ent
roducts
Datamonitor HealthcarePharma intelligence |
10
types of no
carcinomas
cell maligna
leukemia. I
on-small cell
s; and IMG52
ancies such
nitial data fro
lung cancers
29 (Phase I)
as non-Hodg
om these thr
s, ovarian ca
, a CD37-tar
gkin’s lymph
ree compoun
ancers, as we
rgeting TAP c
oma and chr
nds are expe
ell as other
conjugate to
ronic lympho
cted in 2013
treat B-
ocytic
3.
Datamonitor HealthcarePharma intelligence |
11
Medivation
treatment o
docetaxel i
following th
Dimebon (l
(although t
approved w
and since b
impressive
for Xtandi,
part to its s
drug by onc
physicians
Medivation
prechemot
most impor
represente
opportunity
expected in
positive, he
therapy, wh
would resu
for the drug
earlier-stag
that the ong
patients wit
declines of
naïve and c
induce tum
disease rat
’s lead produ
of metastatic
n August 20
he previous f
atrepirdine),
he company
with a hefty w
becoming av
$14m in sal
Astellas. At t
substantial pr
cologists 4 w
reporting po
emphasized
herapy trial,
rtant study fo
d a "gateway
y." This trial,
n the second
elp Xtandi to
hich is Mediv
lt in greater
g, thereby inc
ge clinical da
going Phase
th progressiv
f prostate-spe
chemotherap
mor regressio
te of 49% in
uct Xtandi (e
c castrate-res
12, allowing
failure with its
which led to
y did not allud
wholesale ac
vailable on Se
es, half of w
the conferen
romotional ef
weeks after it
ostchemother
d that the on
PREVAIL, w
or the compa
y to a larger
results for w
d half of 2014
move up to
vation’s ultim
numbers of p
creasing its s
ata with Xtand
e III trial will b
ve CRPC, Xt
ecific antigen
pyexperience
ns with a pa
soft tissues f
enzalutamide
sistant prosta
the compan
s late-stage A
o it being dro
de to this at t
cquisition cos
eptember 13
hich has gon
nce, Medivati
fforts, which
t was launch
rapy details f
going Phase
was now the
any, as it
commercial
which are
4, will, if
earlier lines
mate goal. Th
patients bein
sales potenti
di in the prec
be successfu
tandi was fou
n at all dosag
ed patients. T
rtial respons
for the overa
Medivateyes turprechem
emt
pre
e) was approv
ate cancer (C
y to breathe
Alzheimer’s
opped from M
the conferen
st of $7,450 f
3, the drug ha
ne to the com
ion attributed
led to an 88
ed as well as
for Septemb
e III
of
is
ng eligible
ial. The com
chemotherap
ul. In a Phase
und to induce
ges and in bo
The drug wa
se rate of 22%
all patient pop
tion: with rn to potemotherapy
Medivatimphasizedthe ongo
Phase Iechemoth
trial
ved for the
CRPC) follow
a sigh of rel
disease can
Medivation’s p
ce). Xtandi w
for a 30-day
as brought in
mpany’s co-li
d such high s
8% awarenes
s over 50% o
er.
pany discuss
py setting ind
e I/II trial in 1
e substantial
oth chemoth
as also found
% and stable
pulation.
Xtandi launtial in y setting
ion d that
oing III herapy
wing
ief
didate
pipeline
was
supply,
n an
censor
sales in
ss of the
of
sed how
dicate
40
l
erapy-
d to
e
unched,
Datamonitor HealthcarePharma intelligence |
12
Medivation
leading dru
Zytiga (abir
uptake in p
Medivation
Xtandi. In a
improveme
better than
prechemot
with metas
randomized
twice daily
primary end
statistically
arm of the
prednisone
versus a pr
However, t
since it was
significant
Medivation
including a
who have n
combinatio
discussed
Medivation
2012.
also pointed
ug for metast
raterone; Joh
prechemothe
believes this
addition, if Xt
ent in overall
Zytiga, whic
herapy trial.
tatic CRPC w
d to receive Z
or placebo p
dpoints. Inte
y significant im
study compa
e resulted in
respecified p
the endpoint
s an interim
OS benefit a
went on to d
Phase II tria
not yet been
on trial with X
its plans to e
began enro
d to the patte
tatic CRPC p
hnson & Joh
rapy patients
s indicates th
tandi can de
survival (OS
ch just misse
This trial, kn
who had not
Zytiga 1,000
plus predniso
rim analysis
mprovement
ared to the co
an estimated
p-value of 0.0
that was mis
analysis, and
at the end of
discuss the a
al in hormone
castrated, a
Xtandi in com
expand into a
lling patients
erns of use o
patients who
nson), which
s in the US s
hat a similar
monstrate a
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Datamonitor HealthcarePharma intelligence |
13
NPS Pharm
the treatme
human par
hypoparath
Gattex has
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confident o
outcome of
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unanimous
voted 10 to
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Datamonitor HealthcarePharma intelligence |
14
company’s
request a p
standard 12
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if NPS sub
planned, D
company’s
mid-2014. W
EMA appro
Europe on
approval of
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Datamonitor HealthcarePharma intelligence |
15
Novo Nord
developme
franchise, t
VIII) was fil
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ment option fo
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Datamonitor HealthcarePharma intelligence |
16
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competitive
attributed to
based on J
more globa
duration of
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Datamonitor HealthcarePharma intelligence |
17
Rigel’s Cha
and broad
tyrosine kin
preventing
with inadeq
(DMARDS)
trials (OSK
fourth Phas
compound
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metabolism
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Datamonitor HealthcarePharma intelligence |
18
c
The com2012 perf
will be wdown daggre
competitoof enox
M&A has b
generics ar
bolstering i
generics m
Teva. With
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well as sev
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Datamonitor HealthcarePharma intelligence |
19
2012 has b
aimed at lo
Zyrtec (cet
newer drug
(rotigotine)
evident dur
Keppra (UC
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arthritis dru
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Datamonitor HealthcarePharma intelligence |
20
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well as Pha
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Datamonitor HealthcarePharma intelligence |
21
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the moformRem
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pulmonary
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Datamonitor HealthcarePharma intelligence |
22
Moving to a
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