FM December 2020 digital P

was superior to oral FTC/TDF
tablets.
Lonafarnib for
Hutchinson-
Gilford progeria
The US FDA has approved
lonafarnib (Zokinvy)
capsules for the treatment
of progeria and processingdeficient
progeroid
laminopathies (PL).
The drug has been
approved for reducing the risk
of death from Hutchinson-
Gilford progeria syndrome, as
well as for the treatment of
some PL in patients 1 year and
older.
Progeria is an ultra-rare,
fatal paediatric rapid-aging
disease.
Progeria is caused by a
genetic mutation in the LMNA
(lamin A) gene and results in
a disease-causing abnormal
protein called progerin. There
are approximately 400 children
worldwide with progeria.
Lonafarnib is a
farnesyltransferase inhibitor
(FTI) that has shown a
survival benefit in children
with progeria. Data based
on information from the
PRF International Patient
Registry and clinical trials
co-coordinated by PRF and
Boston Children’s Hospital
demonstrated that in patients
with progeria, lonafarnib
reduced the incidence of
mortality by 60% (p=0.0064)
EMA panel recommends
dabigatran for treating
VTE in children
The European Medicines
Agency’s (EMA)
Committee for Medicinal
Products for Human Use
(CHMP) has adopted a
positive opinion on the
proposed indication for
dabigatran etexilate
(Pradaxa) for the treatment
of venous thromboembolic
events (VTE) and prevention
of recurrent VTE in paediatric
patients from birth to less
than 18 years of age.
If the proposed
indication is approved by
the European Commission
(EC), paediatric patients and
healthcare professionals
will have access to an oral
anticoagulant therapy,
Boehringer Ingelheim said.
At present, there is no
approved therapy for the
treatment or prevention of
blood clots in veins (VTE)
for children, and current
standard of care (SOC) is
associated with a range of
limitations – including the
need for frequent monitoring
of anticoagulation level or
burden of daily injections.
The positive CHMP
opinion is based on a
dedicated paediatric clinical
programme. The DIVERSITY
trial demonstrated that
dabigatran was non-inferior
to SOC for paediatric
patients at high risk of VTE,
with comparable bleeding
rates, while the Brandão
L et al. study showed
favourable safety results
with dabigatran in children
with VTE and persistent
thrombosis risk factors.
Acalabrutinib
to treat adult
patients with
CLL in Europe
AstraZeneca said its
acalabrutinib (Calquence)
has been approved in the
European Union (EU) for the
treatment of adult patients with
chronic lymphocytic leukaemia
(CLL), the most common type
of leukaemia in adults.
Acalabrutinib is a nextgeneration
selective Bruton’s
tyrosine kinase (BTK) inhibitor.
The approval by the
European Commission was
based on positive results from
two phase III clinical trials,
ELEVATE-TN in patients with
previously untreated CLL
and ASCEND in patients with
relapsed or refractory CLL. This
follows a recommendation for
approval by the Committee for
Medicinal Products for Human
Use of the European Medicines
Agency in July 2020.
In the ELEVATE-TN phase
III trial, acalabrutinib combined
with obinutuzumab and as
monotherapy reduced the risk
of disease progression or death
by 90% and 80%, respectively,
compared with standard
chemo-immunotherapy
treatment chlorambucil plus
obinutuzumab, in patients
with previously untreated
and increased average
survival time by
2.5 years. Without
lonafarnib treatment,
children with progeria
die of heart disease at
an average age of 14.5
years.
Patients with the
syndrome treated with
lonafarnib reported
an increased lifespan of
3 months through the
first 3 years of treatment
versus control—and an
increase of 2.5 years through a
maximum follow-up period of
11 years.
Lonafarnib’s approval for
the treatment of certain, rare
processing-deficient progeroid
laminopathies was based
on observed similarities in
underlying genetic mechanisms
of disease and other available
data.
42 / FUTURE MEDICINE / December 2020