Is Pegcetacoplan legal in the United States?

One drug with two unrelated approvals. As Empaveli, a drip under the skin, it treats a rare blood disease called PNH. There it beat the older drug eculizumab, raising haemoglobin by 3.84 g/dL more at 16 weeks. As Syfovre, a shot into the eye, it treats a late form of age-related vision loss. There it slowed the damaged patch from growing by 16 to 22 percent, but sight was no better at 24 months. European regulators refused the eye use for exactly that reason.

Pegcetacoplan is two identical small ring-shaped proteins, joined by a long chain of a plastic-like material called PEG. Most of its weight is the PEG.

How it works: It sticks to C3, and to C3b, a piece of it, and stops C3 being split. That blocks the step shared by all three routes the complement system uses.

Approval history: FDA-approved twice. Empaveli (NDA 215014) was approved 14 May 2021 as a drip under the skin. It is for adults with paroxysmal nocturnal haemoglobinuria, a rare disease where the immune system destroys red blood cells. It is now also approved from age 12 for two rare kidney diseases, C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis. Syfovre (NDA 217171) was approved 17 February 2023 as a shot into the eye. It is for geographic atrophy, a late stage of age-related macular degeneration. The European Medicines Agency refused the geographic atrophy use in 2024.

Approves prescription drugs, each for a named use. It also acts against unapproved products, including peptides sold online. Compounding pharmacies, which mix drugs to order, may make some compounds with a prescription. That only applies when the FDA puts the compound in a category of bulk ingredients it allows.

The legal category above is how The Longevity Archive reads U.S. Food and Drug Administration listings for Pegcetacoplan on the reviewed date. It is not a legal opinion. Rules change. Bringing a compound into the country, prescribing it and owning it can each be treated differently. Check with the agency before you act on anything here.

Check FDA directly

Whether Pegcetacoplan is banned in sport is set by the World Anti-Doping Code and by military rules, not by its legal status in the United States. Before you compete or get tested, look up Pegcetacoplan by name on the Prohibited List, or look up a named product in Global DRO. Under the World Anti-Doping Code, category S0 bans, at all times, any drug-like substance that meets two tests. No other section of the List covers it. And no government health regulator anywhere has a current approval for it as a treatment in people. Many substances that are approved somewhere are banned by name in another section. So approval does not mean a substance is allowed in sport.

• WADA Prohibited List
• Global DRO (check a named product)
• USADA (US athletes)
• Operation Supplement Safety (US service members)

The rules you compete or serve under decide this, not this page.