The Approved Drug: Pegcetacoplan (Empaveli/Syfovre) This is actually a single aptamer molecule with two distinct FDA approvals for different diseases: Paroxysmal Nocturnal Hemoglobinuria (PNH): Approved in 2021 under the brand name Empaveli. It is a complement C3 inhibitor used to treat adults with PNH. Geographic Atrophy (GA): Approved in 2023 under the brand name Syfovre. It is used to treat GA secondary to age-related macular degeneration (AMD). How it works: Pegcetacoplan is a pegylated (attached to a polyethylene glycol chain for longer circulation) aptamer that binds specifically to complement protein C3. By inhibiting C3, it suppresses a central part of the body's complement immune system, which is overactive in both PNH and GA. Important Context and Other Notable Aptamers First-Ever Approval: The first aptamer therapeutic ever approved was Pegaptanib (Macugen) in 2004 for wet AMD. It targets VEGF. While it was a pioneering drug, it has since been largely superseded by more effective antibody-based treatments (like ranibizumab and aflibercept). It is still approved but rarely used. International Approvals: Another significant aptamer is Avacincaptad pegol (Izervay), which was also FDA-approved for Geographic Atrophy in 2023. It works by inhibiting complement C5. Aptamers in Diagnostics: While therapeutic aptamers are rare, aptamers are widely used in research and diagnostic tools. A famous example is the SOMAscan platform, which uses thousands of Slow Off-rate Modified…
