Apellis Pharmaceuticals is targeting Australian approval as a priority, as it seeks to expand the availability of its first-in-class treatment for geographic atrophic age-related macular degeneration (AMD).
At the time of print, Apellis had indicated it would seek to submit an application to the Therapeutic Goods Administration (TGA) in the first quarter of 2023.1
In what’s been described as “the most important event in retinal ophthalmology in more than a decade”, the United States Food and Drug Administration (FDA) approved pegcetacoplan injection (Syfovre) as the first and only treatment for geographic atrophy (GA) secondary to AMD.
AMD is a leading cause of blindness and severe vision loss. Until now there has been no treatment for the ‘dry’ form of the disease.
Welcome News
The US approval for the ground-breaking treatment was welcomed in Australia, with the Macular Disease Foundation Australia (MDFA) describing it as “promising for Australians living with geographic atrophy due to late-stage age-related macular degeneration”.
“An estimated 96,000 people in Australia are currently living with geographic atrophy,” MDFA CEO Dr Kathy Chapman said.
“The approval by the US Food and Drug Administration of pegcetacoplan is a great leap forward for a condition which until now has had zero treatment options and which affects so many older Australians.”
“Like many, we look forward to this new treatment becoming available in Australia,” Dr Chapman told mivision.
Vitreoretinal surgeon and ophthalmologist Associate Professor Andrew Chang, Head of Ophthalmology at Sydney Eye Hospital, took part in the clinical trials as Medical Director of Sydney Retina.
“As the first approved therapy for geographic atrophy, the US FDA approval of Syfovre is a meaningful advance for our patients,” he said.
“As a clinician, seeing the visual impact of geographic atrophy on affected patients and their carers can be devastating.
“As an investigator involved in the clinical trials demonstrating the ability of Syfovre to reduce progression of geographic atrophy, it is wonderful to see the trial results and the benefits extended to the wider population. We look forward to access to this therapy for Australian patients,” Assoc Prof Chang said.
Global Timeframe
In a presentation to investors on the timeframe for a global rollout, Apellis said:
- A marketing authorisation application was under review with the European Medicines Agency,
- A European Commission decision was expected early 2024,
- A marketing application had been submittedin Canada, and
- Submissions to Switzerland, Australia and the United Kingdom were expected in Q1 2023.1
The approval of Syfovre is based on positive results from the Phase 3 OAKS and DERBY studies at 24 months across a broad and representative population of patients, Apellis said in a news release.2
“The approval of Syfovre is the most important event in retinal ophthalmology in more than a decade,” said Dr Eleonora Lad, lead investigator for the OAKS study, director of ophthalmology clinical research, associate professor of ophthalmology, Duke University Medical Center.
Decade in the Making
Apellis CEO and co-founder Dr Cedric Francois said the approval of Syfovre in the United States was “an extraordinary milestone for patients, the retina community, and Apellis”.
“GA is a complex disease that the field has spent decades trying to address, so we are humbled and proud to bring forward the first-ever treatment. Thank you to all who helped make this moment a reality,” he said.
The FDA approval for Syfovre is for GA patients with or without subfoveal involvement and provides dosing flexibility for patients and physicians with a dosing regimen of every 25 to 60 days.
In the OAKS and DERBY studies, Syfovre reduced the rate of GA lesion growth compared to sham and demonstrated increasing treatment effects over time, with the greatest benefit (up to 36% reduction in lesion growth with monthly treatment in DERBY) occurring between months 18–24.
References
- Apellis Pharmaceuticals, Syfovre (pegcetacoplan injection) FDA Approval Conference Call, 17 February 2023 (presentation) available at investors.apellis.com/static-files/140dee61-6cfb-4f96-b421-541dc117f7e8 [accessed 28 Feb 2023].
- Apellis Pharmaceuticals, FDA Approves Syfovre (pegcetacoplan injection) as the First and Only Treatment for Geographic Atrophy (GA), a Leading Cause of Blindness (news release), 17 February 2023, available at investors.apellis.com/news-releases/news-release-details/fda-approves-syfovretm-pegcetacoplan-injection-first-and-only [accessed 28 Feb 2023].
