FM AUGUST 2018 ISSUE1 - digital edition

transduction of Fc-activating receptors
and B-cell receptor, and ultimately
reduces antibody-mediated destruction
of platelets.
The approval was based on data
from two Phase 3 trials, an open-label
extension study, and an initial proof of
concept study. Efficacy was evaluated
in 163 patients with ITP and safety was
evaluated in more than 4,600 patients
across other indications for which
fostamatinib is being studied.
Osimertinib
for 1st-line
NSCLC treatment
T
agrisso (osimertinib) has been
granted approval by US FDA for the
1st-line treatment of patients with
metastatic non-small cell lung cancer
(NSCLC) whose tumours have epidermal
growth factor receptor (EGFR) mutations
(exon 19 deletions or exon 21
L858R mutations).
The approval is based on results from
the phase 3 FLAURA trial, which were
presented at the European Society of
Medical Oncology 2017 Congress and
published in the New England Journal
of Medicine.
The FLAURA trial compared osimertinib
to current 1st-line EGFR tyrosine kinase
inhibitors (TKIs)— erlotinib or gefitinib
— in previously-untreated patients with
locally-advanced or metastatic EGFRmutated
(EGFRm) NSCLC. Osimertinib
met the primary endpoint of progressionfree
survival (PFS). PFS results with
osimertinib were consistent across
all pre-specified patient subgroups,
including patients with or without central
nervous system (CNS) metastases. Overall
survival data were not mature at the time
of the final PFS analysis.
Osimertinib is a third-generation,
irreversible EGFR-TKI designed to inhibit
both EGFR-sensitising and EGFR T790Mresistance
mutations, with clinical activity
against CNS metastases.
Burosumab cleared
for x-linked
hypophosphatemia
T
he US Food and Drug
Administration approved Crysvita
(burosumab) to treat adults and
children of ages 1 year and older with
x-linked hypophosphatemia (XLH), a
rare, inherited form of rickets. XLH
causes low levels of phosphorus in the
blood. It leads to impaired bone growth
and development in children and
adolescents and problems with bone
mineralization throughout a
patient’s life.
Clinical studies found 94 percent of adults
receiving burosumab once a month
achieved normal phosphorus levels
compared to 8 percent of those receiving
placebo. In children, 94 to 100 percent of
patients treated with burosumab every
two weeks achieved normal phosphorus
levels. In both children and adults, X-ray
findings associated with XLH improved
with burosumab therapy.
FDA panel okays
baricitinib for
rheumatoid arthritis
T
he US FDA’s Arthritis Advisory
Committee recommended the
approval of the 2-mg dose of baricitinib,
a once-daily oral medication for the
treatment of moderately-to-severely
active rheumatoid arthritis (RA).
Baricitinib is indicated for adult patients
who have had an inadequate response or
intolerance to methotrexate.
Baricitinib is a once-daily oral JAK
inhibitor currently in clinical studies for
inflammatory and autoimmune diseases.
JAK-dependent cytokines have been
implicated in the pathogenesis of a
number of inflammatory and autoimmune
diseases, suggesting that JAK inhibitors
may be useful for the treatment of a
broad range of inflammatory conditions,
including rheumatoid arthritis.
The Advisory Committee’s
recommendation was based on
baricitinib’s global development
programme, which included four
completed Phase 3 studies in 3,492
patients. The studies evaluated baricitinib’s
treatment impact related to RA signs and
symptoms, physical function, joint damage
progression and other patient-reported
outcomes
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