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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.

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.

Source: Medscape

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.

Source: Medscape

The US FDA has approved Setmelanotide, a melanocortin-4 receptor (MC4R) agonist, is the first FDA-approved therapy for BBS, a rare genetic disorder that impairs a hunger signal along the melanocortin-4 receptor (MC4R) pathway.

Individuals with Bardet-Biedl Syndrome typically have obesity that starts at age 1 along with insatiable hunger (hyperphagia). Available weight management options are generally unsuccessful. Other symptoms may include retinal degeneration, reduced kidney function, or extra digits of the hands or feet.

The most common adverse reactions (with an incidence ≥ 20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection.

Source: Medscape