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  New FDA Approval: Deucravacitinib (Sotyktu), a First-In-Class Oral, Allosteric Tyrosine Kinase 2 Inhibitor

  In September 2022, deucravacitinib (Sotyktu), a first-in-class oral, allosteric tyrosine kinase 2 (TYK2) inhibitor for moderate-to-severe plaque psoriasis, was approved by the FDA. Aside from its first-in-class mechanism of action, it is the first approved de novo deuterated drug. This premium deep dive into deucravacitinib includes: See our overview of all 36 of the novel…

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  An Intracellular Enzyme Drug via Endocytosis: Olipudase Alfa (Xenpozyme)

  olipudase alfa (Xenpozyme), hydrolytic lysosomal sphingomyelin-specific enzyme, Starting dose: 0.1 mg/kg IV q2w (adults); 0.03 mg/kg IV q2w (pediatrics); Maintenance dose: 3 mg/kg IV q2w (adults); 3 mg/kg IV q2w (pediatrics), Non–central nervous system manifestations of acid sphingomyelinase deficiency, Sanofi

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  EMA Approval: Lenacapavir (Sunlenca), a First-In-Class HIV Capsid Inhibitor

  In August 2022, the European Medicines Agency granted Marketing Authorization to lenacapavir (Sunlenca), the first-in-class bi-annually dosed HIV capsid inhibitor indicated for multidrug-resistant HIV-1 infection. This Premium deep dive into Sunlenca includes: See our overview of all 36 of the novel small molecule and large molecule FDA approvals for 2022 here.

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  Billion-Dollar Molecules: Avacopan (Tavneos), a First-in-Class C5aR Antagonist Acquired by Amgen

  Amgen has acquired ChemoCentryx in a deal that includes lead molecule avacopan (Tavneos), a first-in-class orally bioavailable small molecule complement 5a receptor (C5aR) antagonist approved as an adjunctive treatment to standard of care therapy for adults with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV). In this article we review: the details of the deal…

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  Molecule Spotlight: Risdiplam, the First Approved Small Molecule Splicing Modulator for Spinal Muscular Atrophy

  Spinal Muscular Atrophy (SMA)—the leading genetic cause of childhood mortality—is a disease in which the gene SMN1 is deleted or mutated. The highly similar gene SMN2 can undergo alternative splicing to generate functional protein transcripts of the SMN1 gene product, although native levels of the protein are too low to overcome the pathology. Here we…

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  FDA Novel Drug Approvals: May 2022

  There were two novel small molecules approved in May 2022: And one novel large molecule approval: This deep dive explores each drug in more detail, including the industry context, the clinical data, the target rationale, mechanisms of action, and drug origins. See our overview of all 36 of the novel small molecule and large molecule…