Pegcetacoplan has launched already in the United States and Europe for PNH, a rare hematological disease. As one of the few drugs to have completed phase 3 trials for GA, pegcetacoplan is anticipated to be the first drug to launch for GA or “dry late age-related macular degeneration (AMD),” which has no approved pharmacotherapy. The U.S. Food and Drug Administration (FDA)’s granting of fast track status and priority review designation underscores the unmet need in this underserved patient population and the potential for pegcetacoplan to experience commercial success upon launch.
Subcutaneous pegcetacoplan generated $15.1 million in sales in 2021 following its launch in the United States in May 2021 to treat PNH. Additional global approvals for PNH will likely contribute to greater sales over the coming years, and the contribution of sales for PNH is likely to be smaller than that for GA.
If launched when expected, pegcetacoplan will be the first GA therapy to market. Its novel mechanism of action targets complement C3, which has been detected in drusen in the retina of GA patients and shown to induce VEGF expression. It has demonstrated the ability to delay GA progression, and its potential bimonthly dosing could prove to be an advantage over other competitors will monthly dosing only.
The New Drug Application (NDA) submitted to the U.S. FDA for GA was supported by results from phase 2 and 3 trials:
December 2021:
For adults with PNH who are anemic after treatment with a C5 inhibitor for at least three months:
• Marketing authorization: European Commission (EC)
For GA secondary to AMD:
July 2018:
• Priority Review designation: U.S. FDA
July 2022:
• Fast track designation: U.S. FDA
December 2022:
• Anticipated submission for marketing authorization: European Medicines Agency (EMA)
February 2023:
• PDUFA date (FDA)
Actual and expected launch:
PNH:
• 2021: United States
• 2022: Europe
GA:
• 2023: United States
• 2024: Europe
Patents estimated to expire beginning in 2033
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