chemotherapy in the trial went longer
(median 8.2 months) without certain
complications (failure to achieve complete remission within 60 days of starting treatment, progression of leukemia or
death) than patients who received chemotherapy alone (median three months).
Rydapt was also approved for adults
with certain types of rare blood disorders
(aggressive systemic mastocytosis, systemic mastocytosis with associated hema-tological neoplasm or mast cell leukemia).
BioMarin Pharmaceutical received ap-
proval from the FDA for Brineura (
cerliponase alfa) as a treatment for a specific
form of Batten disease. Brineura is the first
FDA-approved treatment to slow loss of
walking ability in symptomatic pediatric
patients 3 years of age and older with late
infantile neuronal ceroid lipofuscinosis
type 2 (CLN2), also known as tripeptidyl
peptidase-1 (TPP1) deficiency.
CLN2 disease is one of a group of disorders known as neuronal ceroid lipofus-cinoses (NCLs), collectively referred to as
Batten disease, a rare inherited disorder
that primarily affects the nervous system.
The initial symptoms usually include language delay, recurrent seizures (epilepsy)
and difficulty coordinating movements.
This condition often requires the use of
a wheelchair by late childhood and they
typically do not survive past their teens.
Batten disease is relatively rare, occurring in an estimated two to four of every
100,000 live births in the U.S.
Brineura is an enzyme replacement
therapy. Its active ingredient (cerliponase
alfa) is a recombinant form of human
TPP1, the enzyme deficient in patients
with CLN2 disease.
The efficacy of Brineura was established
in a non-randomized, single-arm dose escalation clinical study in 22 symptomatic
pediatric patients with CLN2 disease and
compared to 42 untreated patients with
CLN2 disease from a natural history cohort who were at least 3 years old and had
motor or language symptoms. Taking into
account age, baseline walking ability and
genotype, Brineura-treated patients demonstrated fewer declines in walking ability compared to untreated patients in the
natural history cohort.
The safety of Brineura was evaluated in
24 patients with CLN2 disease aged 3 to
8 years who received at least one dose of
Brineura in clinical studies.
PIQUR Therapeutics lead compound,
PQR309 for the treatment of patients
with diffuse large B-cell lymphoma
(DLBCL), has been granted orphan drug
designation by the European Medicines
DLBCL is an aggressive form of lymphoma, and the most common type of
non-Hodgkin lymphoma (NHL), accounting for about 30 percent of all NHL
cases. The disease occurs primarily in older individuals, though it can also occur in
children and young adults in rare cases.
In addition to this orphan drug designation by the EMA in DLBCL, PIQUR
has also recently received orphan drug
designation from the FDA for PQR309
for the treatment of primary CNS lymphoma (PCNSL).
The EMA orphan drug designation
is assigned to a medicine intended for
use against a rare condition and allows a
pharmaceutical company to benefit from
incentives offered by the EU to develop
a medicine for the treatment, prevention or diagnosis of a disease that is life
threatening or a chronically debilitating
Novartis received approval from the
FDA for Kisqali Femara Co-Pack (ribociclib
tablets; letrozole tablets) for the treatment
of hormone receptor-positive, human
epidermal growth factor receptor-2 negative (HR+/HER2-) advanced or metastatic
breast cancer in postmenopausal women.
The Kisqali Femara Co-Pack is the first,
and only currently available, combination
pack with two prescription products in advanced breast cancer.
The packaging of the Kisqali Femara
Co-Pack allows patients the convenience
of obtaining a full 28-day cycle of the two
medicines in one package with one prescription and one co-pay.
The Kisqali Femara Co-Pack is available in three dosage strengths: Kisqali 600
mg plus Femara 2.5 mg, Kisqali 400 mg
plus Femara 2.5 mg, and Kisqali 200 mg
plus Femara 2.5 mg.
Kisqali was approved on March 13,
2017 in combination with an aromatase
inhibitor as initial endocrine-based ther-
apy for the treatment of postmenopaus-
al women with HR+/HER2- advanced
or metastatic breast cancer. Femara is
an aromatase inhibitor approved for
first-line treatment of postmenopausal
women with HR+ or unknown advanced
EMD Serono, the biopharmaceutical
business of Merck KGaA, Darmstadt,
Germany, and Pfizer received approval
from the FDA for BAVENCIO (avelumab)
Injection for the treatment of patients
with locally advanced or metastatic urothelial carcinoma (UC) who have disease
progression during or following chemotherapy, or adjuvant treatment with
chemotherapy. BAVENCIO was previously granted accelerated approval from
the FDA for the treatment of adults and
pediatric patients 12 years and older with
metastatic Merkel cell carcinoma (MCC).
These indications are approved under
accelerated approval based on tumor response and duration of response.
Bladder cancer makes up approximately 90% of urothelial carcinomas and
is the sixth most common cancer in the
U.S. When the disease has metastasized,
the five-year survival rate is approximately
5%. Despite advances in the treatment of
locally advanced or metastatic urothelial
carcinoma, the prognosis for patients remains poor and more treatment options
The efficacy and safety of BAVENCIO was demonstrated in the urothelial
carcinoma cohorts of a Phase I study of
BAVENCIO in the treatment of various
solid tumors. The urothelial carcinoma
cohorts enrolled patients with locally
advanced or metastatic urothelial carcinoma with disease progression on or
after platinum-containing chemotherapy
or who had disease progression within
12 months of treatment with a platinum-containing neoadjuvant or adjuvant chemotherapy regimen.
BAVENCIO is designed to potentially
engage both the adaptive and innate immune systems. By binding to PD-L1,
BAVENCIO is thought to prevent tumor
cells from using PD-L1 for protection
against white blood cells, such as T cells,
exposing them to anti-tumor responses.
BAVENCIO has also been shown to induce antibody-dependent cell-mediated
cytotoxicity (ADCC) in vitro. CP