CLINICAL NEWS
safety and efficacy results from a clinical
trial of 29 adults with acquired hemophilia A who received Obizur to treat
a serious bleeding episode. The drug
demonstrated treatment efficacy and
there were no safety concerns identified. Acquired hemophilia A is a rare,
but potentially life-threatening, bleeding
disorder caused by the development of
antibodies (immune system proteins)
directed against the body’s own FVIII, a
protein important for blood clotting. The
recombinant analogue of porcine (pig)
FVIII contained in Obizur is similar
enough to human FVIII to be effective
in blood clotting, but is less likely to be
affected by the antibodies against human
FVIII that are present in people with
acquired hemophilia A.
Source: FDA News Release. Accessed from www.fda.gov/NewsEvents/
Newsroom/PressAnnouncements/ucm420263.htm
New Sickle Cell
Disease Treatment
Receives Fast Track
Designation
NKTT120, a humanized monoclonal
antibody that specifically deletes iNKT
cells designed to treat sickle cell disease
(SCD), was recently granted the FDA’s
“Fast Track” designation. iNKT cells are
regulatory T cells that have been demonstrated to act as key mediators of organ
damage in preclinical models of SCD. Fast
Track designation is intended to facilitate
development and expedite review of drugs
meant to treat serious or life-threatening
medical conditions, as well as drugs that
demonstrate the potential to address
unmet medical needs. NKTT120 previously received the FDA’s “Orphan Drug”
designation for the treatment of SCD.
T:7”
T:10”
Lenalidomide caused thalidomide-type limb defects in monkey offspring.
General disorders and administration site conditions: Chills
If this drug is used during pregnancy, or if the patient becomes pregnant
Musculoskeletal and connective tissue disorders: Pain in extremity
while taking this drug, the patient should be apprised of the potential
Nervous system disorders: Dysguesia, headache, neuropathy peripheral
hazard to a fetus.
Infections and infestations: Respiratory tract infection, sinusitis,
nasopharyngitis
If pregnancy does occur during treatment, immediately discontinue the
Skin and subcutaneous tissue disorders: Dry skin, night sweats
drug. Under these conditions, refer patient to an obstetrician/gynecologist
experienced in reproductive toxicity for further evaluation and counseling.
The following serious adverse events not described above and reported in
Any suspected fetal exposure to REVLIMID must be reported to the FDA
2 or more patients treated with REVLIMID monotherapy for mantle cell
via the MedWatch program at 1-800-FDA-1088 and also to Celgene
lymphoma.
Corporation at 1-888-423-5436.
Respiratory, Thoracic and Mediastinal Disorders: Chronic obstructive
pulmonary disease
Animal data
Infections and Infestations: Clostridium difficile colitis, sepsis
In an embryo-fetal developmental toxicity study in monkeys, teratogenicity,
Neoplasms benign, malignant and unspecified (incl cysts and polyps):
including thalidomide-like limb defects, occurred in offspring when pregnant
Basal cell carcinoma
monkeys received oral lenalidomide during organogenesis. Exposure
Cardiac Disorder: Supraventricular tachycardia
(AUC) in monkeys at the lowest dose was 0.17 times the human exposure
at the maximum recommended human dose (MRHD) of 25 mg. Similar
6.4 Postmarketing Experience
studies in pregnant rabbits and rats at 20 times and 200 times the MRHD
The following adverse drug reactions have been identified from the
respectively, produced embryo lethality in rabbits and no adverse
worldwide post-marketing experience with REVLIMID: Allergic conditions
reproductive effects in rats.
(angioedema, SJS, TEN), tumor lysis syndrome (TLS) and tumor flare
reaction (TFR), pneumonitis, hepatic failure, including fatality, toxic hepatitis,
In a pre- and post-natal development study in rats, animals received
cytolytic hepatitis, cholestatic hepatitis, and mixed cytolytic/cholestatic
lenalidomide from organogenesis through lactation. The study revealed
hepatitis and transient abnormal liver laboratory tests. Because these
a few adverse effects on the offspring of female rats treated with
reactions are reported voluntarily from a population of uncertain size, it
lenalidomide at doses up to 500 mg/kg (approximately 200 times the
is not always possible to reliably estimate Z\