This section highlights FDA-related milestones and regulatory updates for drugs developed by Apellis Pharmaceuticals (APLS).
Over the past two years, Apellis Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
pegcetacoplan and Pegcetacoplan. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
pegcetacoplan - FDA Regulatory Timeline and Events
pegcetacoplan is a drug developed by Apellis Pharmaceuticals for the following indication: Paroxysmal nocturnal hemoglobinuria (PNH).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- pegcetacoplan
- Announced Date:
- June 6, 2025
- Indication:
- Paroxysmal nocturnal hemoglobinuria (PNH)
Announcement
Apellis Pharmaceuticals, Inc today presented new data from the open-label period of the Phase 3 VALIANT study, investigating EMPAVELI® (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
AI Summary
Apellis Pharmaceuticals, Inc. recently presented new data from the open-label period of its Phase 3 VALIANT study. This study investigated EMPAVELI® (pegcetacoplan) for treating C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). Researchers found that patients treated with EMPAVELI experienced a remarkable 68% reduction in proteinuria at Week 26 compared to placebo, and this improvement was maintained through one year. In addition, patients maintained stable kidney function as measured by estimated glomerular filtration rate (eGFR).
The results highlight the potential of EMPAVELI to address key markers of these rare and severe kidney diseases while demonstrating a safety profile with no new safety signals. These findings support EMPAVELI’s promise in offering substantial clinical benefits and a meaningful treatment option for patients in need.
Read Announcement- Drug:
- pegcetacoplan
- Announced Date:
- April 1, 2025
- Indication:
- Paroxysmal nocturnal hemoglobinuria (PNH)
Announcement
Apellis Pharmaceuticals, announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation of the supplemental New Drug Application (sNDA) for EMPAVELI® (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are severe and rare kidney diseases.
AI Summary
Apellis Pharmaceuticals announced that the FDA has accepted and granted Priority Review designation for the supplemental New Drug Application (sNDA) of EMPAVELI® (pegcetacoplan). This designation applies to the treatment of severe and rare kidney diseases: C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). With this Priority Review, the FDA will focus on evaluating EMPAVELI more quickly, and the target approval action date is July 28, 2025. The sNDA is supported by positive results from the Phase 3 VALIANT study, where EMPAVELI showed clinically meaningful benefits including a significant reduction of protein in urine, stabilization of kidney function, and a notable decrease in C3c staining. If approved, EMPAVELI could offer a potentially life-changing treatment option for patients at high risk of progressing to kidney failure.
Read Announcement- Drug:
- pegcetacoplan
- Announced Date:
- April 1, 2025
- Estimated Event Date Range:
- July 28, 2025 - July 28, 2025
- Target Action Date:
- July 28, 2025
- Indication:
- Paroxysmal nocturnal hemoglobinuria (PNH)
Announcement
Apellis Pharmaceuticals, Inc. announced that The Prescription Drug User Fee Act (PDUFA) target action date is July 28, 2025.
AI Summary
Apellis Pharmaceuticals, Inc. recently announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for EMPAVELI® with Priority Review designation. The key target action date under the Prescription Drug User Fee Act (PDUFA) is July 28, 2025. This designation is for the treatment of two rare and severe kidney diseases: C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
The move follows promising Phase 3 VALIANT study results that showed EMPAVELI’s ability to reduce proteinuria, stabilize kidney function, and significantly lower C3c staining in affected patients. If approved, EMPAVELI could offer a disease-modifying therapy for patients at high risk of progression to kidney failure, potentially making a life-changing difference in care and treatment outcomes.
Read Announcement- Drug:
- pegcetacoplan
- Announced Date:
- January 27, 2025
- Indication:
- Paroxysmal nocturnal hemoglobinuria (PNH)
Announcement
Apellis Pharmaceuticals, announced that the Therapeutic Goods Administration (TGA) has approved SYFOVRE® (pegcetacoplan) for the every-other-month treatment of adult patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) with an intact fovea and when central vision is threatened by GA lesion growth.
AI Summary
Apellis Pharmaceuticals has received approval from Australia’s Therapeutic Goods Administration (TGA) for its treatment SYFOVRE® (pegcetacoplan). This new therapy is designed for adults with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) who still have an intact fovea, especially when GA lesion growth threatens central vision. Patients will receive the treatment every other month, marking SYFOVRE as the first approved therapy for GA in Australia.
The approval is based on clinical trial data showing that SYFOVRE can slow the progression of the disease, which is a major cause of irreversible vision loss. This milestone is expected to bring hope and improved quality of life for many Australians affected by GA, offering a new way to manage a condition that has limited treatment options until now.
Read Announcement- Drug:
- pegcetacoplan
- Announced Date:
- June 28, 2024
- Indication:
- Paroxysmal nocturnal hemoglobinuria (PNH)
Announcement
Apellis Pharmaceuticals, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the marketing authorization application (MAA) of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA).
AI Summary
Apellis Pharmaceuticals recently announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a negative opinion on the marketing authorization application for intravitreal pegcetacoplan, a treatment designed for geographic atrophy (GA). This outcome was marked by several dissenting votes from CHMP members, reflecting mixed opinions during the review process.
The decision followed an Ad Hoc Expert Group meeting where experts agreed that the GA lesion size is a suitable primary outcome for GA trials and highlighted microperimetry as the best functional measure available. Despite the setback, Apellis remains committed to GA patients in Europe and plans to seek a re-examination of the decision. The company expects a final opinion on the treatment in the fourth quarter of 2024, as it continues its efforts to bring this treatment option to patients suffering from the irreversible vision loss caused by GA.
Read Announcement- Drug:
- pegcetacoplan
- Announced Date:
- May 24, 2024
- Indication:
- Paroxysmal nocturnal hemoglobinuria (PNH)
Announcement
Apellis Pharmaceuticals, Inc announced positive one-year results from the Phase 2 NOBLE study investigating systemic pegcetacoplan, a targeted C3 therapy, for the treatment of post-transplant recurrence of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
AI Summary
Apellis Pharmaceuticals recently announced encouraging one-year results from its Phase 2 NOBLE study, exploring systemic pegcetacoplan for treating post-transplant recurrence of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). The study showed that pegcetacoplan rapidly reduced disease activity within 12 weeks, with these improvements maintained over the long term. At one year, 64% of treated patients had a significant decrease in C3c deposits, with 55% showing zero C3c staining intensity, indicating a complete clearance of these deposits. These findings suggest that pegcetacoplan targets the underlying disease process by reducing harmful complement activity, which can lead to kidney inflammation and damage. The positive safety profile and sustained improvements in proteinuria and kidney function further support the potential of pegcetacoplan as a promising treatment for these patients.
Read Announcement
Pegcetacoplan (VALIANT) - FDA Regulatory Timeline and Events
Pegcetacoplan (VALIANT) is a drug developed by Apellis Pharmaceuticals for the following indication: Primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G), two rare and debilitating kidney diseases.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Pegcetacoplan (VALIANT)
- Announced Date:
- August 8, 2024
- Indication:
- Primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G), two rare and debilitating kidney diseases
Announcement
Apellis Pharmaceuticals, Inc. announced positive topline results from the Phase 3 VALIANT study investigating systemic pegcetacoplan in patients with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are rare kidney diseases with no approved treatments.
AI Summary
Apellis Pharmaceuticals announced positive topline data from its Phase 3 VALIANT study, which evaluated systemic pegcetacoplan in patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), two rare kidney diseases with no approved treatments. The study met its primary endpoint, showing a statistically significant 68% reduction in proteinuria compared with placebo. These results were consistent across all subgroups—including adolescents and adults as well as patients with native and post-transplant kidneys—and confirmed the drug’s robust safety profile.
Encouraged by these findings, the companies plan to submit a supplemental new drug application to the U.S. Food and Drug Administration and pursue regulatory approval in the European Union. This progress represents a promising step toward addressing critical unmet needs for patients battling these severe kidney conditions.
Read Announcement
Free APLS Stock Alerts