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Industry leaders react to FDA’s pegcetacoplan approval

Article

Ophthalmologists see new therapy as having a major impact on how geographic atrophy is treated.

The FDA on Friday announced its approval of the pegcetacoplan injection (SYFOVRE, Apellis Pharmaceuticals Inc.) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

Pegcetacoplan, according to Apellis, is the first and only FDA-approved treatment for GA. The approval was based on the results of the 24-month phase 3 data submitted in its New Drug Application in November 2022.

“For the first time ever, we are celebrating the approval of a treatment for GA,” said Jeff Todd, president and chief executive officer, Prevent Blindness. “This is a historic and hopeful day for all GA patients and their care partners, who have been waiting for a treatment for this relentless form of vision loss.”

For ophthalmologists, the approval is welcome news and offers a new treatment option for patients.

Peter J. McDonnell, MD

Peter J. McDonnell, MD

Peter J. McDonnell, MD, director of the Wilmer Eye Institute and chief medical editor of Ophthalmology Times, pointed out that it was a major advance to have a therapy for neovascular AMD and now (considering the much larger number of patients with atrophic AMD) this represents an even greater achievement in terms of addressing unmet need.

“The availability of this new therapy will have major implications in terms of the ability of us ophthalmologists to treat this extremely large number of patients and for payors (especially Medicare) to absorb this additional cost,” McDonnell said.

Rishi P. Singh, MD, president of Cleveland Clinic Martin North and South hospitals in Stuart, Florida, noted that pegcetacoplan is a first-in-class therapy for GA secondary to AMD.

Rishi P. Singh, MD

Rishi P. Singh, MD

“It is a value to patients and providers of care,” he said. “With regards to what it can do to slow the progression of this disease state over time. It obviously will be taken and monitored with its efficacy and safety together as the real world analysis are started. And I think we are going learn a lot in the first year of treatment as to what this drug holds for our patients and holds for their potential progression of geographic atrophy over time.”

Carl Regillo, MD, director of retina services at Wills Eye Hospital and a professor of ophthalmology at Thomas Jefferson University in Philadelphia, touted the approval.

Carl Regillo, MD

Carl Regillo, MD

“The FDA approval of a therapy to reduce the rate of growth of geographic atrophy is a landmark event,” Regillo said. “The benefit of a course of pegcetacoplan is a relatively small but significant step forward in managing an otherwise relentlessly progressive, visually disabling, endstage form of dry AMD that was previously untreatable.”

Andrew A. Moshfeghi, MD, MBA

Andrew A. Moshfeghi, MD, MBA

According to Andrew A. Moshfeghi, MD, MBA, medical director, USC Roski Eye Institute, the approval of pegcetacoplan for the treatment of geographic atrophy secondary to AMD “signifies more about the FDA's commitment to therapies that result in preservation of photoreceptor function and less about the therapeutic benefit of the treatment, which is nominal.”

“The FDA recognizes that this commitment is fundamental to advancing our understanding of the disease process and therapeutic targets that can slow down, subsequently halt, and ultimately reverse GA with potential functional gain,” he said. 

Moshfeshi recounted that the path to ophthalmology’s current therapies of wet AMD was paved by the destructive laser photocoagulation of photoreceptors followed by the less damaging photodynamic therapy and then by pegaptamer-based anti-VEGF therapy to the current intravitreal pan-anti-VEGF therapies which actually deliver visual acuity gain—a therapeutic development arc spanning 40 years.

“We hope to shorten that drug development timeline in dry AMD with the current approval, which represents the first small step in that process,” he said.

Sharon Fekrat, MD

Sharon Fekrat, MD

Sharon Fekrat, MD, a retinal surgeon and director of the Duke Vitreoretinal Surgery Fellowship Program, also lauded the FDA’s decision.

“Not only have I been anticipating a positive FDA decision for many months but so have many of my patients with geographic atrophy,” she said. “Thus far, these patients have had no treatment options and have had to live with the real looming threat of central vision loss. This is a historic and welcome decision for everyone on the front lines of this relentless disease."

Ian C. Han, MD

Ian C. Han, MD

Ian C. Han, MD, an associate professor, Institute for Vision Research, Ophthalmology and Visual Sciences, University of Iowa Hospital and Clinics, pointed out that counseling patients who have lost vision due to geographic atrophy can be very challenging because of the lack of available treatment options for dry AMD.

“I often tell patients that treating dry AMD is easier said than done because the "A" in AMD stands for "age" -- the challenge is slowing down aging, and finding effective ways to slow aging has been a human quest that has spanned the centuries (think of the epic explorations to find the mythical Fountain of Youth,” he said.

Moreover, Han also noted that it has been known for some time that complement dysregulation plays a critical role in the pathophysiology of AMD. Pegcetacoplan targets a key component of complement (C3) that is known to be activated in AMD, including those with genetic risk for AMD (e.g., CFH variants).

“The approval of pegcetacoplan is exciting because it is the first FDA-approved treatment for GA secondary to dry AMD,” he said. “But to balance the enthusiasm, I tell patients that the drug is not a ‘fountain of youth,’ and that the results in clinical trial were measurable but modest.”

Han also noted that, as with any new drug, ophthalmologists will have to translate aspects from clinical trials into a "real-world" setting.

“Although available data suggest a reasonable safety profile, there was a signal toward increased incidence of wet AMD in treated patients relative to sham, and this deserves further exploration,” he said. “The route of drug delivery is via intravitreal injection, which is convenient and familiar to both doctors and patients.”

Pragmatically, however, Han explained that many practices are already strained with a high injection burden for wet AMD, and will need to adapt clinic workflows to accommodate an increase due to patients receiving dry AMD treatment as well.

Han also noted that pegcetacoplan is designed to slow the rate of GA progression in dry AMD, which is a huge step in AMD treatment.

“Notably, the drug had a measurable effect in clinical trials on slowing GA growth (i.e., an anatomic/structural endpoint), but functional measures including visual acuity were not different between patients treated with sham,” he concluded. “As such, the day-to-day impact of slowing GA growth in patients remains to be seen. For patients who have already lost central vision due to foveal GA, restorative therapies (e.g., stem cell transplantation) currently in development are perhaps the next frontier and the next hope.”

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