Apellis receives approval for intravitreal pegcetacoplan for GA

Pegcetacoplan is the first FDA-approved treatment for geographic atrophy (GA) secondary to age-related macular degeneration.

March 3, 2023

Syfovre™ (intravitreal pegcetacoplan) has been FDA approved for geographic atrophy (GA) secondary to age-related macular degeneration. Apellis’s pegcetacoplan already has FDA approval as a subcutaneous infusion, under the brand name Empaveli®, for the treatment of paroxysmal nocturnal hemoglobinuria.  

Drug name: Syfovre™ (intravitreal pegcetacoplan)
Manufacturer: Apellis 
Condition: Geographic atrophy secondary to age-related macular degeneration 

Condition overview 

Geographic atrophy is an advanced form of age-related macular degeneration that leads to progressive and irreversible vision loss. It is a leading cause of blindness that impacts one million people in the U.S.  

GA is caused by atrophy of retinal cells which causes lesion growth. As lesions progress and expand, central vision is severely impaired. It can affect one or both eyes, but a patient with GA in one eye is more likely to develop it in the other. Approximately 50% of patients with GA in one eye will demonstrate bilateral GA within seven years of diagnosis. GA is a leading cause of impaired visual function in the elderly.1 

Risk factors for GA include: 

  • Genetic polymorphisms  
  • Advanced age (especially over 85 years old)  
  • Smoking  
  • Presence of early AMD to GA in the other eye 

GA progression causes a gradual loss of visual function. Symptoms include scotomas (large dark or blind spots in the visual field), difficulty recognizing faces, decreased reading speed (measured in words per minute, wpm), impaired dark adaptation, low luminance deficit (LLD), impaired contrast sensitivity, and difficulty driving at night.1 

Current treatment 2-6 

There are no FDA approved treatments for GA. Vascular endothelial growth factor inhibitors [e.g., Lucentis® (ranibizumab), Eylea® (aflibercept)] have not been found effective in treating GA. 

Intravitreal pegcetacoplan overview 2-6 

The intravitreal pegcetacoplan New Drug Application to the FDA included two Phase 3 randomized, double-blind, placebo-controlled trials and one Phase 2 randomized, single-masked, placebo-controlled trial. Patients in the Phase 3 trials received one injection in one eye either every month or every other month. For patients that had bilateral GA (~80%), the injection was given in the worst eye. The primary endpoint in both phase 3 trials was the change in the total area of GA lesions from baseline at 12 months. The studies showed reduction in GA lesion growth compared with placebo. No clinically meaningful difference was observed between the pegcetacoplan and placebo group in visual function. But Apellis notes that this was consistent with expectations as visual function loss occurs over longer periods of time. 

PDUFA: February 26, 2023 
Benefit: Medical benefit, intravitreal  

Prime monitors the drug pipeline 

The drug pipeline is full of new, groundbreaking specialty drugs. Prime focuses on clinical strategies designed to keep clients ahead of drug trends — because it’s easier to manage change when you see it coming.  

Robust extended GA pipeline 7-8 

Drug name 

Manufacturer 

Drug Class 

Route of Administration 

Potential FDA decision 

avacincaptad pegol 

Iveric Bio 

PEGylated anti-C5 aptamer 

Intravitreal 

2023 

ALK-001 

Alkeus 

Chemically modified vitamin A 

Orally 

2024 

NGM621 

NGM Biopharmaceuticals 

Humanized IgG1 monoclonal antibody designed to inhibit C3 

Intravitreal 

 

2026 

galegenimab 

Genentech/Roche 

Novel antibody directed against high-temperature requirement protein A1 (HtrA1) 

Intravitreal injection 

2026 

IONIS-FB-LRx 

Ionis Pharmaceuticals 

Specific antisense oligonucleotide that targets the complement factor B gene 

SC 

2026 

elamipretide 

Stealth Biotherapeutics 

Tetrapeptide targeting cardiolipin in mitochondria 

IV/SC 

2026 

GT005 

Gyroscope 

AAV-based gene therapy expressing complement factor I 

Subretinal one-time treatment 

2027 

 References 

  1. Geographic Atrophy. Accessed at: https://www.geographicatrophy.org/ 
  2. Amylyx Pharmaceutcals announces FDA acceptance and Priority Review of New Drug Application (NDA) for AMX0035 for the treatment of ALS. Accessed in April 2022 at: https://www.biospace.com/article/releases/amylyx-pharmaceuticals-announces-fda-acceptance-and-priority-review-of-new-drug-application-nda-for-amx0035-for-the-treatment-of-als/ 
  3. Liao DS, Grossi FV, Mehdi DE, et al. Complement C3 inhibitor pegcetacoplan for geographic atrophy secondary to age-related macular degeneration. Ophthalmology 2020; 127 (2): 186-195.  
  4. Apellis announces top-line results from Phase 3 DERBY and OAKS studies in GA and plans to submit NDA to FDA in the first half of 2022. Accessed in September 2022 at: https://investors.apellis.com/news-releases/news-release-details/apellis-announces-top-line-results-phase-3-derby-and-oaks 
  5. Apellis announces 240month results showing increased effects over time with pegcetacoplan in Phase 3 DERBY and OAKS studies in GA. Accessed in October 2022 at: https://investors.apellis.com/news-releases/news-release-details/apellis-announces-24-month-results-showing-increased-effects 
  6. Apellis. Data on file. October 2022. 
  7. Geographic atrophy treatment in the pipeline for 2022. Accessed in October 2022 at: https://www.ophthalmologyadvisor.com/slideshow/retina-vitreous/potential-therapies-for-geographic-atrophy-in-amd/ 
  8. Cortellis. Search term: geographic atrophy. Accessed October 2022. 

 

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