Drugs of today
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Uterine leiomyomas represent the most common form of benign gynecological tumors affecting 20-40% of women during their life. Several therapeutic options are available for treating these patients. The use of medical treatment for myomas has largely grown in the last years, in particular for women who would refuse, postpone or are not candidates for surgery. ⋯ Relugolix is an orally active nonpeptide GnRH-ant, recently licensed for marketing in Japan for the treatment of symptoms related to uterine myomas. Currently, several phase III clinical trials are ongoing to evaluate this molecule in this setting in the U. S. and Europe.
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Erdafitinib is the first Food and Drug Administration (FDA)-approved oral pan-fibroblast growth factor receptor (FGFR) kinase inhibitor that binds to four FGFRs (FGFR-1 to -4), leading to decreased cell signaling and cellular apoptosis. Erdafitinib also binds to RET, colony-stimulating factor 1 receptor (CSF-1R), platelet-derived growth factor receptor α and β (PDGFR-α and PDGFR-β), Fms-related tyrosine kinase 4 (FLT4), KIT and vascular endothelial growth factor receptor 2 (VEGFR-2), exhibiting additional antitumor mechanisms resulting in cell kill. ⋯ S. for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma with susceptible FGFR3/FGFR2 genetic alterations following progression during or after at least one line of platinum-containing chemotherapy and within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. Erdafitinib is the first oral treatment option for patients with urothelial carcinoma.
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Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare and life-threatening disease characterized by microangiopathic hemolytic anemia, thrombocytopenia and multiorgan failure, resulting from autoantibody-mediated severe A disintegrin and metalloproteinase with thrombospondin motifs 13 (ADAMTS13) deficiency. In spite of treatment with plasma exchange and immunosuppression, patients remain at risk of exacerbations, refractoriness and death. Caplacizumab (Cablivi; Ablynx, a Sanofi company), a nanobody targeting von Willebrand factor (vWF), has been recently approved in the E. ⋯ We detail here the preclinical and clinical data on caplacizumab for iTTP, including the recent studies that led to approval by the U. S. Food and Drug Administration (FDA) in 2019.
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Atherosclerotic cardiovascular disease is the leading cause of death all over the world. Its etiopathogenesis involves many correlated processes, with hypercholesterolemia being one of the main risk factors. Several large clinical trials have established the association between low-density lipoprotein cholesterol (LDL-C) levels and cardiovascular events. ⋯ Statins are the cornerstone of pharmacological lipid-lowering treatment, although they are not always successful in attaining the recommended LDL-C levels. Therefore, newer and more potent therapies have been developed, being prominent among them ezetimibe and especially the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors. Recent trials with these new therapies have reaffirmed the theory of 'the lower, the better' when it comes to LDL-C levels, and 'the earlier, the better' when it comes to atherosclerotic physiopathology.
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Epilepsy is one of the most common chronic disorders of the brain affecting around 70 million people worldwide. Treatment is mainly symptomatic, and most patients achieve long-term seizure control. Up to one-third of the affected subjects, however, are resistant to anticonvulsant therapy. ⋯ Cannabidiol (CBD) is the first in a new class of antiepileptic drugs. It is a major chemical of the cannabis plant, which has antiseizure properties in absence of psychoactive effects. This article provides a critical review of the pharmacology of CBD and the most recent clinical studies that evaluated its efficacy and safety as adjunctive treatment of seizures associated with LGS and DS.