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.
Select a button below to view the list of FDA events for that drug.
Pegcetacoplan (VALIANT) FDA Regulatory 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:
- December 3, 2025
- Indication:
- Primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G), two rare and debilitating kidney diseases
Announcement
Apellis Pharmaceuticals, announced that The New England Journal of Medicine (NEJM) published positive results from the Phase 3 VALIANT study investigating 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 reported that The New England Journal of Medicine published positive Phase 3 VALIANT results for EMPAVELI® (pegcetacoplan) in C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), two rare, serious kidney diseases. VALIANT was a randomized, placebo-controlled, double-blind trial of 124 patients aged 12 and older, including adolescents, adults, and patients with C3G recurring after transplant. The study evaluated outcomes after 26 weeks, with urine protein-to-creatinine ratio (UPCR) as the primary endpoint.
EMPAVELI showed robust benefit across three key markers: a 68% reduction in proteinuria versus placebo (p<0.0001), stabilization of kidney function with a +6.3 mL/min/1.73 m2 eGFR difference (nominal p=0.03), and marked clearance of C3 deposits—71% of treated patients achieved zero C3 staining (nominal p<0.0001). Safety and tolerability were favorable and consistent with the known profile; common adverse reactions included infusion-site reactions, fever, nasopharyngitis, influenza, cough, and nausea. Results were consistent across age groups and transplant status.
Read Announcement- 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
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 approved by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- pegcetacoplan
- Announced Date:
- November 12, 2025
- Indication:
- Paroxysmal nocturnal hemoglobinuria (PNH)
Announcement
Apellis Pharmaceuticals, announced data from a post hoc analysis of the GALE extension study following five years of continuous treatment with SYFOVRE® (pegcetacoplan injection), the leading treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
AI Summary
Apellis Pharmaceuticals reported a post hoc analysis of the GALE extension study after five years of continuous treatment with SYFOVRE (pegcetacoplan). Both every‑other‑month and monthly dosing delayed geographic atrophy (GA) lesion growth by about 1.5 years in patients with nonsubfoveal GA compared with sham/projected sham.
GALE is a Phase 3, multicenter, open‑label extension (n=792) that followed patients from the OAKS and DERBY trials who remained on the same regimen. Because many sham patients switched to treatment after Month 24, a projected sham arm—based on observed linear two‑year lesion growth and validated by a fellow‑eye analysis—was used to estimate untreated progression through Year 5.
The company said the safety profile through five years remained consistent with earlier reports. Detailed results are planned for presentation at a future medical meeting.
Investigators noted these findings suggest early, continuous treatment with SYFOVRE can meaningfully delay GA progression and may alter the disease course.
Read Announcement- Drug:
- pegcetacoplan
- Announced Date:
- October 20, 2025
- Indication:
- Paroxysmal nocturnal hemoglobinuria (PNH)
Announcement
Apellis Pharmaceuticals, announced new data from the open-label period of the Phase 3 VALIANT study that reinforce the robust and sustained efficacy of EMPAVELI® (pegcetacoplan), a C3 inhibitor, in patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare and debilitating kidney diseases.
AI Summary
Apellis released new data from the open-label period of the Phase 3 VALIANT study showing that EMPAVELI® (pegcetacoplan) delivers strong, lasting benefits in patients with C3 glomerulopathy and primary IC-MPGN. Over one year, EMPAVELI maintained a 68% reduction in proteinuria versus placebo, regardless of immunosuppressant use or baseline proteinuria levels. One-third of treated patients achieved full proteinuria remission (UPCR ≤ 0.5 g/g) by week 26, compared with just 3% on placebo, and these gains held through week 52. The treatment also stabilized kidney function (eGFR) and continued to show a well-understood safety profile with no new concerns.
Two indirect treatment comparisons using data from the VALIANT study and the Phase 3 APPEAR-C3G iptacopan trial found EMPAVELI superior in lowering proteinuria and reaching a combined endpoint of proteinuria reduction and eGFR stability. These analyses suggest EMPAVELI may offer better kidney protection than iptacopan in C3G patients, supporting its role as the only approved therapy for these rare diseases in patients 12 and older.
Read Announcement- Drug:
- pegcetacoplan
- Announced Date:
- July 28, 2025
- Indication:
- Paroxysmal nocturnal hemoglobinuria (PNH)
Announcement
Apellis Pharmaceuticals, announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. C3G and primary IC-MPGN are rare kidney diseases, affecting 5,000 people in the United States.1
AI Summary
Apellis Pharmaceuticals announced FDA approval of EMPAVELI® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged 12 and older. These rare kidney diseases affect about 5,000 people in the U.S. and often lead to kidney failure.
In the Phase 3 VALIANT trial, EMPAVELI achieved a 68% reduction in proteinuria, stabilized kidney function, and cleared C3 deposits versus placebo. Benefits were seen in adolescents, adults, and patients with post-transplant disease recurrence. The broad approval is backed by a well-established safety profile from over 2,200 patient-years of data. EMPAVELI offers a new option to reduce proteinuria and preserve kidney function for those living with these challenging diseases.
Read Announcement- Drug:
- pegcetacoplan
- Announced Date:
- July 15, 2025
- Indication:
- Paroxysmal nocturnal hemoglobinuria (PNH)
Announcement
Apellis Pharmaceuticals, announced that five abstracts were accepted for oral presentation at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting to be held July 30 - August 2 in Long Beach, California.
AI Summary
Apellis Pharmaceuticals announced that five abstracts have been accepted for oral presentation at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting. The meeting will take place from July 30 to August 2, 2025, in Long Beach, California. These presentations will highlight important clinical insights related to SYFOVRE® (pegcetacoplan injection) and its use in treating geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Experts will discuss data that demonstrate SYFOVRE’s effectiveness in slowing the progression of GA, showcasing its strong safety profile and benefits even with as few as six doses per year. Dr. Caroline Baumal, Chief Medical Officer at Apellis, emphasized the significant interest from retina specialists in this innovative treatment, highlighting its potential to improve patient outcomes and offer hope for those affected by this progressive vision-threatening disease.
Read Announcement- 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
- 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:
- 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:
- 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
Free APLS Stock Alerts