FM December 2020 digital P

drug approvals
Gene therapy for
methylmalonic
acidemia gets
orphan drug status
Asklepios BioPharmaceutical
and Selecta Biosciences
announced that the US FDA has
granted orphan drug designation
to MMA-101, an adeno-associated
virus (AAV)-based gene therapy
in development for the treatment
of isolated methylmalonic
acidemia (MMA) due to methyl
malonyl-CoA mutase (MMUT)
gene mutations.
MMA-101 previously
received rare paediatric disease
designation from the FDA in
October 2020.
MMA is a rare monogenic
disorder in which the body
cannot break down certain
proteins and fats. This metabolic
disease may lead to metabolic
crisis and is associated with
long-term complications,
including feeding problems,
developmental delays,
intellectual impairment,
chronic kidney disease, optic
nerve atrophy, osteopenia
and pancreatitis. Typically,
well-managed patients have
periods of relative health
with intermittent metabolic
decompensation events that
may result in multiorgan failure,
triggered by intercurrent
infections or stress episodes.
Symptoms of MMA usually
appear in early infancy and vary
from mild to life-threatening.
Without treatment, this disorder
can lead to coma and, in some
cases, death.
AskBio and Selecta expect
to initiate a phase 1 clinical trial
of MMA-101 and ImmTOR for
patients with MMA in the first half
of 2021.
Additional
dosing option
for durvalumab
in NSCLC
AstraZeneca’s durvalumab
(Imfinzi) has been
approved in the US for an
additional dosing option, a
1,500mg fixed dose every
four weeks, in the approved
indications of unresectable
Stage III non-small cell
lung cancer (NSCLC) after
chemoradiation therapy
(CRT) and previously treated
advanced bladder cancer.
This new option is
consistent with the approved
durvalumab dosing in
extensive-stage small cell
lung cancer (ES-SCLC) and
will be available to patients
weighing more than 30kg as
an alternative to the approved
weight-based dosing of 10mg/
kg every two weeks.
The approval by the US
FDA was based on data from
several durvalumab clinical
trials, including the PACIFIC
phase III trial which supported
the two-week, weight-based
dosing in unresectable Stage
III NSCLC, and the CASPIAN
phase III trial which used fourweek,
fixed-dosing during
maintenance treatment in ES-
SCLC.
The four-week 1,500mg
fixed-dosing option for
durvalumab is also under
regulatory review in several
other countries, including in
the EU where the new dosing
option was granted accelerated
assessment.
Durvalumab is also
approved for previously treated
patients with advanced bladder
cancer in the US and several
other countries. Additionally,
it is approved in the US, the
EU, Japan and several other
countries around the world
for the treatment of ES-SCLC
based on the CASPIAN phase
III trial.
Durvalumab is a human
monoclonal antibody that
binds to PD-L1 and blocks
the interaction of PD-L1 with
PD-1 and CD80, countering
the tumour’s immune-evading
40 / FUTURE MEDICINE / December 2020