FM December 2020 digital P

homologous recombination
deficient (HRD)-positive
advanced ovarian cancer.
The approval by the
European Commission
was based on a biomarker
subgroup analysis of the
PAOLA-1 phase III trial
which showed olaparib, in
combination with bevacizumab
maintenance treatment,
demonstrated a substantial
progression-free survival
(PFS) improvement versus
bevacizumab alone for
patients with HRD-positive
advanced ovarian cancer. It
follows the recommendation
for approval by the Committee
for Medicinal Products for
Human Use of the European
Medicines Agency in
September 2020.
The PAOLA-1 phase III
trial showed that olaparib, in
combination with bevacizumab
maintenance treatment,
reduced the risk of disease
progression or death by 67%
(based on a hazard ratio of
0.33; 95% confidence interval
0.25-0.45). The addition of
olaparib improved PFS to a
median of 37.2 months versus
17.7 with bevacizumab alone
in patients with HRD-positive
advanced ovarian cancer.
Olaparib with bevacizumab
provided benefit beyond first
disease progression, improving
PFS2 to a median of 50.3
months versus 35.3 with
bevacizumab alone.
The full EU indication is
for olaparib in combination
with bevacizumab for the
maintenance treatment of
adult patients with advanced
(FIGO Stages III and IV) highgrade
epithelial ovarian,
fallopian tube or primary
peritoneal cancer who are
in response (complete or
partial) following completion
of 1st-line platinum-based
chemotherapy in combination
with bevacizumab and
whose cancer is associated
with HRD positive status
defined by either a breast
cancer susceptibility gene 1/2
(BRCA1/2) mutation and/or
genomic instability.
Olaparib is a first-in-class
PARP inhibitor and the first
targeted treatment to block
DNA damage response (DDR)
in cells/tumours harbouring
a deficiency in homologous
recombination repair (HRR),
such as mutations in BRCA1
and/or BRCA2.
Perampanel
for paediatric
epilepsy
E
isai Co received approval
from the European
Commission for the use of
its anti-epileptic agent (AED)
perampanel (Fycompa) in
the treatment of paediatric
patients.
This approval extends
the use of perampanel
as adjunctive therapy for
partial-onset seizures (POS)
(with or without secondary
generalization) by expanding
the approved age range from
12 years and above to 4 years
and above, and for primary
generalized tonic-clonic
seizures (PGTCS) from 12 years
and above to 7 years and
above.
The approval was
based on the results of
phase III (Study 311) and
phase II (Study 232) clinical
studies conducted globally
to evaluate perampanel
as adjunctive therapy in
paediatric patients with POS
or PGTCS. Study 311 evaluated
the safety, tolerability, and
exposure-efficacy relationship
of perampanel when
administered as adjunctive
therapy in paediatric patients
aged 4 to less than 12 years
with inadequately controlled
POS or PGTCS.
This study showed that
the safety and efficacy of the
perampanel combination
therapy in paediatric epilepsy
patients with poorly controlled
partial seizures (ages 4 to less
than 12 years) were similar to
those in patients aged 12 years
and older.
PLX-300 gets
rare paediatric
desig to
treat GM2
gangliosidosis
Polaryx Therapeutics, Inc
announced that it has
received from the USFDA both
Rare Pediatric Disease and
Orphan Drug designations
for the treatment of GM2
gangliosidosis with PLX-300.
GM2 gangliosidosis,
also known as Tay-Sachs
and Sandhoff diseases,
are ultra-rare and fatal
paediatric neurodegenerative
disorders caused by defects
in Hexosaminidase A (HEXA)
and Hexosaminidase B (HEXB),
key enzymes in the lysosome,
respectively.
PLX-300 is a novel, small
molecule found in many plants
as a deaminated product of
44 / FUTURE MEDICINE / December 2020