Navepegritide is described as the first therapy to deliver continuous C-type natriuretic peptide exposure over a weekly dosing interval in eligible pediatric patients.
The FDA approved Itvisma, the first gene replacement therapy for children 2 years and older, teens, and adults with spinal muscular atrophy, a one-time treatment that replaces the SMN1 gene and demonstrated improved motor function in Phase 3 studies.