A Newly Approved Alkyne: Covalent FGFR1-4 Kinase Inhibitor, Futibatinib
Futibatinib (Lytgobi) is a rare alkyne-containing, irreversible FGFR1-4 inhibitor that received accelerated approval from the FDA in September 2022. Structurally distinct from other approved FGFR2/3 inhibitors, futibatinib treats a range of FGFR-aberrant tumors, including those with fusions or TKI-resistant mutants like the V565I/L gatekeeper mutation. This Premium report reviews: See our overview of all 36…
A Prodrug Bioactivated in the Eye for Ocular Hypertension: Omidenepag
Omidenepag isopropyl (Omlonti) is a prodrug of a prostaglandin E2 (EP2) receptor agonist that reduces pressure in the eye. Upon topical administration to the eye, the molecule penetrates the lipophilic and negatively charged corneal epithelium and is converted to the pharmacologically active form by the enzyme carboxylesterase-1. The selectivity of the molecule for EP2 over…
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…
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
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.
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…