Tezepelumab is an epithelial cytokine, and is the first and only biologic approved in Europe by EC for severe asthma for adults and adolescents with inadequately controlled severe asthma with no phenotype or biomarker limitations. Tezepelumab acts by blocking thymic stromal lymphopoietin (TSLP). Most common adverse events in patients were pharyngitis, rash, arthralgia, and injection site reactions.
The five patients all of whom had an aggressive form of SLE underwent a single infusion of the experimental treatment. All five patients were able to stop their standard treatments for as long as 17 months following the therapy, the study found. The patients also stopped experiencing severe symptoms such as lung inflammation, fibrosis of the heart valves, arthritis, and fatigue. The patients have not relapsed. All of the patients were treated with genetically engineered T cells known as chimeric antigen receptor (CAR) T-cell therapy, a treatment regularly used to kill cancer cells. Researchers harvested the patients’ immune cells and engineered them to destroy dysfunctional cells when infused back into the body.
RSV is the leading cause of lower respiratory tract infections in infants. Worldwide, acute lower respiratory infections associated with RSV account for about 1.4 million hospitalizations & 27,300 in-hospital deaths among infants under the age of 6 months annually, according to the WHO. Nirsevimab is a long-acting antibody given as a single intramuscular injection at a dose of 50 mg for infants with a body weight of less than 5 kg, and 100 mg for infants weighing at least 5 kg. If approved, nirsevimab will be the only preventative option for the broad newborn and infant population against RSV.
The US FDA has approved terlipressin (Terlivaz), the first and only drug approved for patients with hepatorenal syndrome (HRS). HRS is characterized by a progressive deterioration in kidney function in people with advanced liver disease. Terlipressin is an injectable synthetic vasopressin analogue indicated for patients with HRS who are experiencing rapid deterioration of kidney function (type 1 HRS).
The most commonly observed adverse reactions that occurred in at least 4% of patients treated with terlipressin were abdominal pain (19.5%), nausea (16%), respiratory failure (15.5%), diarrhea (13%), and dyspnea (12.5%).
The US FDA has approved daxibotulinumtoxinA-lanm injection (Daxxify) to temporarily improve the appearance of moderate to severe glabellar lines (frown lines) in adults.
Action: It’s an acetylcholine release inhibitor and neuromuscular blocking agent, is the first peptide-formulated, long-acting neuromodulator approved for this indication.
Adverse effects: headache (6%), eyelid ptosis (2%), and facial paresis, including facial asymmetry (1%).
Contraindicated in adults with hypersensitivity to any botulinum toxin preparation.
The US FDA has approved the gene therapy betibeglogene autotemcel (Zynteglo) for adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. Functional copies of the mutated gene are inserted into patient’s hematopoietic stem cells via a replication-defective lentivirus. A one-time gene therapy cost $2.8 million. Adverse events such as abdominal pain, hot flush, dyspnea, tachycardia, noncardiac chest pain, and cytopenias were noted.
A novel zoonotic RNA Langya virus (LayV) is a henipavirus first detected in the China provinces of Shandong and Henan. The name of the virus refers to Mt. Langya.
Symptoms: fever, fatigue, cough
Langya henipavirus affects humans, dogs, goats, and its presumed original host are shrews.
Lab findings in patients: thrombocytopenia, leukopenia, impaired liver and kidney function test.
Treatment: No specific drug or vaccination only supportive care.
Monkeypox is a zoonotic orthopoxvirus.
Usual onset: 5–21 days post-exposure.
Duration: 2 to 4 weeks.
Route: Human-to-human transmission, exposure to infected body fluids or contaminated objects by small droplets through the airborne route.
Symptoms: Fever, headache, muscle pains, shivering, mostly nonitchy blistering rash on face, hands, soles and swollen lymph nodes.
Complications: Secondary infections, eye infection, visual loss, scarring, encephalitis, sepsis, bronchopneumonia.
Types: Central African (Congo Basin), West African
Diagnosis: Testing for viral DNA PCR
Differential diagnosis: Chickenpox, smallpox
Prevention: Smallpox vaccine, hand washing, covering rash, PPE
Treatment: Supportive, antivirals, vaccinia immune globulin
Medication: Tecovirimat
The US FDA approves crizotinib (Xalkori) for the treatment of unresectable, recurrent, or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors (IMT) in adults and children over 1 year of age.
Crizotinib is a selective tyrosine kinase inhibitor currently approved for the treatment of metastatic non-small cell lung cancer in patients whose tumors are positive for ALK or ROS1 as detected by an FDA-approved test, and for ALD-positive anaplastic large cell lymphoma.
Adverse reactions occurring in 35% or more pediatric patients included vomiting, nausea, diarrhea, abdominal pain, rash, vision disorder, upper respiratory tract infection, cough, pyrexia, musculoskeletal pain, fatigue, edema, constipation, and headache.
The US FDA has approved dabrafenib + trametinib for adult and pediatric patients aged 6 years and older with unresectable or metastatic solid tumors with the BRAF V600E mutation whose disease has progressed following prior treatment and who have no satisfactory alternative treatment options.
The combination represents “the first and only BRAF/MEK inhibitor to be approved with a tumor-agnostic indication for solid tumors carrying the BRAF V600E mutation, which drives tumor growth in more than 20 different tumor types, and it is the only BRAF/MEK inhibitor approved for use in pediatric patients.
The combination already carries indications for BRAF-mutated melanoma, non–small cell lung cancer, and thyroid cancer. The new approval was based on two adult trials and one pediatric trial that showed an overall response benefit for patients with high- and low-grade gliomas, biliary tract cancers, and certain gynecologic and gastrointestinal cancers.
