FDA Approves Syfovre: First Treatment for Geographic Atrophy
Syfovre has received FDA approval as the first-ever treatment for geographic atrophy, marking a significant advancement in managing this debilitating condition.
The FDA approved pegcetacoplan (Syfovre) on February 17, 2023, as the first treatment for geographic atrophy secondary to age-related macular degeneration. The intravitreal C3 inhibitor, developed by Apellis Pharmaceuticals, demonstrated statistically significant reductions in GA lesion growth in the Phase 3 DERBY and OAKS trials.
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Key Takeaways
- FDA approved Syfovre (pegcetacoplan) on February 17, 2023, under NDA 217171—the first therapy for geographic atrophy FDA approval letter
- Phase 3 trials showed 19-22% reduction in GA lesion growth over 24 months with monthly dosing PubMed
- Administered as 15 mg/0.1 mL intravitreal injection monthly or every other month
- Approximately 1 million Americans have geographic atrophy, with no prior FDA-approved treatments
- Commercial availability began in early March 2023 through specialty pharmacies
What Is Geographic Atrophy?
Geographic atrophy is an advanced form of age-related macular degeneration characterized by progressive degeneration of retinal cells, leading to irreversible vision loss. The condition affects approximately 1 million Americans and represents a leading cause of blindness in older adults. Prior to pegcetacoplan's approval, no FDA-approved treatments existed for GA, leaving patients with only supportive care options as their vision deteriorated.
How Was Syfovre Approved?
The FDA approval was based on data from two Phase 3 clinical trials: DERBY (NCT03525600) and OAKS (NCT03525613). These randomized, double-masked, sham-controlled studies enrolled a combined 1,258 patients with geographic atrophy secondary to AMD.
Key efficacy results at 24 months demonstrated:
- OAKS monthly: 22% reduction in GA lesion growth (-0.90 mm², p<0.0001)
- OAKS every-other-month: 18% reduction (-0.74 mm², p=0.0002)
- DERBY monthly: 19% reduction (-0.75 mm², p=0.0004)
- DERBY every-other-month: 16% reduction (-0.63 mm², p=0.0030)
The treatment effect increased over time, with greater reductions observed between months 18-24 compared to earlier periods.
What Is the Safety Profile?
The most common adverse events observed in clinical trials included:
- Ocular injection site reactions (15%)
- Conjunctival hemorrhage (13%)
- Increased intraocular pressure (9%)
- New-onset exudative AMD (12% with monthly dosing, 6% with every-other-month)
Serious ocular treatment-emergent adverse events occurred in 2% of monthly-treated patients, 1% of every-other-month patients, and 1% of sham-treated patients.
How Is the Drug Administered?
Syfovre is administered as an intravitreal injection (15 mg/0.1 mL) by an eye care professional. The approved dosing regimens are:
| Regimen | Injection Frequency | 24-Month Efficacy |
|---|---|---|
| Monthly | Every 4 weeks | 19-22% lesion growth reduction |
| Every-other-month | Every 8 weeks | 16-18% lesion growth reduction |
What Does This Mean for Patients?
For the approximately 1 million Americans living with geographic atrophy, Syfovre represents the first FDA-approved option to slow disease progression. While the treatment does not restore lost vision, it offers the potential to preserve remaining sight for longer.
Dr. Jeffrey S. Heier, principal investigator of the DERBY study and Director of the Retinal Service at Ophthalmic Consultants of Boston, stated: "The approval of pegcetacoplan represents a paradigm shift in how we treat geographic atrophy. For the first time, we have the ability to slow the relentless progression of this disease."
When Will Syfovre Be Available?
Apellis Pharmaceuticals announced that Syfovre would be commercially available in early March 2023 through specialty distributors and specialty pharmacies. The company has established a patient support program to help eligible patients access the therapy.
Frequently Asked Questions
What is geographic atrophy?
Geographic atrophy is an advanced form of age-related macular degeneration characterized by progressive degeneration of retinal cells, leading to irreversible vision loss. It affects approximately 1 million Americans and previously had no FDA-approved treatments.
How does Syfovre work?
Syfovre (pegcetacoplan) is a C3 inhibitor that targets excessive activation of the complement cascade, a key driver of geographic atrophy progression. By inhibiting C3, the drug slows retinal cell death.
How effective is Syfovre in clinical trials?
In the Phase 3 DERBY and OAKS trials, monthly Syfovre reduced GA lesion growth by 19-22% over 24 months compared to sham treatment. Every-other-month dosing reduced lesion growth by 16-18%.
How is Syfovre administered?
Syfovre is administered as an intravitreal injection (15 mg/0.1 mL) by an eye care professional either monthly or every other month, based on the healthcare provider's assessment.
Primary Sources
- FDA Approval Letter for Syfovre (pegcetacoplan) Injection - NDA 217171. U.S. Food and Drug Administration. February 17, 2023.
- Efficacy and Safety of Pegcetacoplan in Geographic Atrophy: Results from the DERBY and OAKS Trials. PubMed. October 2023.
- DERBY Trial (NCT03525600). ClinicalTrials.gov.
- OAKS Trial (NCT03525613). ClinicalTrials.gov.
- Syfovre Orange Book Entry. FDA Orange Book.
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