March 2019 digital v1

progressive damage to any
organ in the body.
Benralizumab is a
monoclonal antibody that binds
directly to IL-5 receptor on
eosinophils and attracts natural
killer cells to induce rapid and
near-complete depletion of
eosinophils via apoptosis.
Benralizumab was
developed by AstraZeneca with
MedImmune.
Dacomitinib to
treat NSCLC
in Europe
The Committee for Medicinal
Products for Human Use
(CHMP) of the European
Medicines Agency (EMA) has
adopted a positive opinion
recommending dacomitinib
45 mg (Vizimpro) for the firstline
treatment advanced or
metastatic non-small cell lung
cancer (NSCLC) with epidermal
growth factor receptor (EGFR)-
activating mutations, according
to Pfizer.
Dacomitinib is an oral,
once-daily, irreversible panhuman
epidermal growth
factor receptor kinase inhibitor.
Dacomitinib was approved
by the US FDA in 2018 for the
first-line treatment of patients
with metastatic NSCLC with
EGFR exon 19 deletion or
exon 21 L858R substitution
mutations as detected by an
FDA-approved test. It was also
recently approved in Japan for
EGFR gene mutation-positive,
inoperable or recurrent NSCLC
The Marketing
Authorization Application
(MAA) for dacomitinib
was based on results from
ARCHER 1050, a randomized,
multicentre, multinational,
open-label, phase 3 study
conducted in patients with
locally advanced unresectable,
or metastatic NSCLC harbouring
EGFR exon 19 deletion or
exon 21 L858R substitution
mutations, an Eastern
Cooperative Oncology Group
(ECOG) performance status of
0 or 1; with no prior therapy for
metastatic disease or recurrent
disease with a minimum of
12 months disease-free after
completion of systemic therapy.
Pexidartinib to
treat TGCT gets
priority review
Daiichi Sankyo said
pexidartinib for the
treatment of tenosynovial giant
cell tumour (TGCT) has been
granted priority review by US
FDA.
TGCT, also referred to
as pigmented villonodular
synovitis (PVNS) or giant cell
tumour of the tendon sheath
(GCT-TS), is a non-malignant
tumour of the joint or tendon
sheath, which can be locally
aggressive and debilitating in
some patients. There are no
currently approved systemic
therapies for TGCT.
The NDA is based on results
of the pivotal phase 3 ENLIVEN
study of oral pexidartinib, the
first placebo-controlled study
of a systemic investigational
therapy in patients with TGCT.
Pexidartinib is an
investigational oral small
molecule that potently inhibits
CSF1R (colony stimulating
factor-1 receptor), which is
a primary growth driver of
abnormal cells in the synovium
that cause TGCT. Pexidartinib
also inhibits c-kit and FLT3-ITD.
Nanobody drug
for aTTP
The US FDA cleared
caplacizumab-yhdp
(Cablivi), an anti-vWF
Ospemifene for vaginal
dryness
The US FDA has expanded
use of ospemifene
(Osphena) to include
treatment of moderate to
severe vaginal dryness due
to menopause.
Vaginal dryness and
itching are symptoms of
vulvar and vaginal atrophy.
Ospemifene is an
oestrogen receptor
nanobody, in combination
with plasma exchange and
immunosuppression for
the treatment of acquired
thrombotic thrombocytopenic
purpura (aTTP) in adults.
Caplacizumab is the
first FDA approved therapy
specifically indicated for the
treatment of aTTP.
Caplacizumab targets von
Willebrand factor (vWF), a
protein in the blood involved
in hemostasis. It is designed to
inhibit the interaction between
vWF and platelets.
agonist/antagonist with
tissue selective effects.
It is available in 60 mg
tablets which were initially
approved in 2013 to
treat moderate to severe
dyspareunia due to
menopause.
The approval has been
on the basis of safety
and efficacy data from
a confirmatory phase
3 randomized, doubleblind,
placebo-controlled
multicentre study evaluating
ospemifene in patients with
moderate to severe vaginal
dryness.
The data showed that
treatment with ospemifene
led to a statistically
significant improvement
in self-reported, most
bothersome symptom of
vaginal dryness as well as
a statistically significant
increase in the proportion
of superficial cells and a
corresponding statistically
significant decrease in the
proportion of parabasal cells
on a vaginal smear; mean
reduction in vaginal pH
between baseline and week
12 was also statistically
significant.
Caplacizumab is Sanofi›s
first Nanobody-based medicine
to receive approval in the US.
Nanobodies are a novel
class of proprietary therapeutic
proteins based on singledomain
antibody fragments
that contain the unique
structural and functional
properties of naturallyoccurring
heavy chain only
antibodies.
The treatment received
FDA Fast Track designation and
was evaluated under Priority
Review.
March 2019 / FUTURE MEDICINE / 35