FM December 2020 digital P

tactics and releasing the
inhibition of immune responses.
Orphan drug
status to
AMX0035 to
treat Wolfram
syndrome
The US FDA granted orphan
drug designation to
AMX0035 for the treatment
of Wolfram syndrome, Amylyx
Pharmaceuticals announced.
Wolfram syndrome is
an autosomal recessive
neurodegenerative disease.
Common manifestations of
Wolfram syndrome include
diabetes mellitus, optic nerve
atrophy, central diabetes
insipidus, sensorineural
deafness, neurogenic bladder,
and progressive neurologic
difficulties.
Genetic and experimental
evidence suggest that
endoplasmic reticulum
(ER) dysfunction is a critical
pathogenic component
of Wolfram syndrome.
The prognosis of Wolfram
syndrome is poor, and many
patients die prematurely with
severe neurological disabilities.
AMX0035 is an
investigational product
designed to reduce neuronal
death and dysfunction.
It targets endoplasmic
reticulum and
mitochondrialdependent
neuronal
degeneration
pathways in ALS and other
neurodegenerative diseases
Cabotegravir
for HIV preexposure
prophylaxis
ViiV Healthcare announced
that the US FDA has
granted Breakthrough Therapy
Fast track designation to
rilzabrutinib for treating ITP
The US FDA has granted Fast
Track Designation (FTD) to the
oral investigational Bruton’s tyrosine
kinase (BTK) inhibitor, rilzabrutinib
for the treatment of immune
thrombocytopenia (ITP).
Designation for long-acting,
injectable cabotegravir for
HIV pre-exposure prophylaxis
(PrEP).
The Breakthrough Therapy
Designation was based on
efficacy and safety results
from HPTN 083, a phase IIb/
III randomised, multicentre,
double-blind, clinical trial
that compared long-acting,
injectable cabotegravir to daily
oral emtricitabine/tenofovir
disoproxil fumarate 200 mg
and 300 mg (FTC/TDF) for
HIV prevention among men
who have sex with men and
transgender women who have
sex with men.
The final analysis of HPTN
083 showed the superiority
of long acting cabotegravir,
which was 66% more effective
In addition, following positive
phase 1/2 study results, a phase 3
study evaluating rilzabrutinib for ITP
has been initiated.
Rilzabrutinib received an orphan
drug designation from the US FDA for
the treatment of ITP in October 2018.
ITP is characterized by immunemediated
platelet destruction and
impairment of platelet production,
which leads to downstream
thrombocytopenia, a predisposition
to bleeding, and adverse impact on
patient quality of life.
BTK is involved in innate and
adaptive immune responses and is
a signalling molecule in immunemediated
diseases. Rilzabrutinib
data demonstrate an ability to block
inflammatory immune cells, eliminate
autoantibody destructive signalling,
and prevent new autoantibody
production without depleting B cells.
at preventing HIV when
compared to daily oral FTC/TDF
tablets. This translated to an
HIV incidence rate of 0.41% in
the cabotegravir group (95%
confidence interval [CI] 0.22%-
0.69%) and 1.22% in the FTC/
TDF group (95% CI 0.87%-
1.67%) in a study population of
4,566. The results of HPTN 083
were presented at the 23rd
International AIDS Conference
(AIDS 2020) in July.
The blinded phase of HPTN
084, a partner HIV prevention
study in sub-Saharan African
women, was stopped earlier
this month based upon the
recommendation of the
independent data safety
monitoring board (DSMB)
following demonstration that
long-acting cabotegravir
December 2020 / FUTURE MEDICINE / 41