Drugs of today
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Porphyrias are a family of rare diseases chiefly due to inborn errors of heme biosynthesis. The porphyrias are generally characterized either by the main site of overproduction of heme precursors (hepatic or erythropoietic) or the main clinical manifestations (acute or cutaneous). The regulation of 5- (or δ)-aminolevulinic acid synthase 1 (ALAS1) plays a key role in the pathway of normal hepatic heme synthesis, providing insight into the pathophysiologic mechanisms and potential therapeutic targets for the treatment of the porphyrias. ⋯ S. Food and Drug Administration (FDA) for the treatment of adult patients with AHP, and it received also approval in the E. U. in 2020 for the treatment of AHP in adults and adolescents aged 12 years and older.
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Ebola virus (EBOV) causes outbreaks of lethal febrile illness in Africa, the largest of which resulted in over 11,000 deaths and represented a global public health threat. A new biomedical countermeasure, the recombinant vesicular stomatitis virus expressing EBOV glycoprotein (rVSV-EBOV) has been licensed (Ervebo; Merck & Co.). rVSV-EBOV is a replicative viral vaccine engineered to express EBOV antigen. ⋯ The vaccine has a good safety profile, but is associated with self-limited arthritis and rash in a minority of recipients. rVSV-EBOV is highly immunogenic after a single intramuscular dose with antibody titers persisting for at least 2 years. In the recent outbreak in the Democratic Republic of the Congo, rVSV-EBOV was administered to more than 300,000 individuals and may have contributed, at least in part, to controlling the epidemic.