WALTHAM, MA, USA I December 16, 2022 I Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced that the company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency for intravitreal pegcetacoplan, an investigational, targeted C3 therapy, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). GA is a leading cause of blindness that impacts more than five million people globally.1,2
“Along with the upcoming FDA decision in the U.S., the submission of our EU marketing application brings us closer than ever toward our goal of delivering pegcetacoplan to patients living with GA around the world,” said Jeffrey Eisele, Ph.D., chief development officer at Apellis. “If approved, pegcetacoplan would be the first and only treatment for GA in Europe.”
“Pegcetacoplan is the only GA treatment to demonstrate increased effects over 24 months across a broad patient population,” said Frank G. Holz, DERBY and OAKS study investigator and professor and chairman of the department of ophthalmology at the University of Bonn, Germany. “There are currently no treatments to slow the progression of GA, a life-altering disease that causes substantial vision loss, so the potential approval of pegcetacoplan would be a significant milestone for patients in Europe.”
The MAA submission is based on results from the Phase 3 DERBY and OAKS studies at 24 months. In the studies, treatment with both every-other-month and monthly pegcetacoplan demonstrated robust and clinically meaningful reductions of GA lesion growth, with treatment effects that increased over time, and a favorable safety profile.
The U.S. marketing application is under review with a Prescription Drug User Fee Act (PDUFA) target action date of February 26, 2023.
About DERBY and OAKS
DERBY (n=621) and OAKS (n=637) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of pegcetacoplan with sham injections across a broad and representative population of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary objective of the studies is to evaluate the efficacy of pegcetacoplan in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence (p-value less than .05) at 12 months. Patients continued to receive masked treatment for 24 months.
About Geographic Atrophy (GA)
Geographic atrophy (GA) is an advanced form of age-related macular degeneration (AMD) and a leading cause of blindness that impacts more than five million people worldwide, including one million people in the United States.1,2 This progressive disease can severely impair visual function, independence, and quality of life as it takes on average 2.5 years for GA lesions to encroach the fovea, which is responsible for central vision.3 GA is caused by destruction of retinal cells through irreversible lesion growth that is driven by excessive complement activation.4 C3 is the only target that can precisely control the complement cascade due to its central location. There are currently no approved treatments for GA.
About Pegcetacoplan for Geographic Atrophy (GA)
Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy.
About Apellis
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in complement, we ushered in the first new class of complement medicine in 15 years with the approval of the first and only targeted C3 therapy. We are advancing this science to continually develop transformative medicines for people living with rare, retinal, and neurological diseases. For more information, please visit http://apellis.com or follow us on Twitter and LinkedIn.
1 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta analysis. Ophthalmology 2012;119:571–580.
2 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116.
3 Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.
4 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261.
SOURCE: Apellis
Post Views: 58
WALTHAM, MA, USA I December 16, 2022 I Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced that the company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency for intravitreal pegcetacoplan, an investigational, targeted C3 therapy, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). GA is a leading cause of blindness that impacts more than five million people globally.1,2
“Along with the upcoming FDA decision in the U.S., the submission of our EU marketing application brings us closer than ever toward our goal of delivering pegcetacoplan to patients living with GA around the world,” said Jeffrey Eisele, Ph.D., chief development officer at Apellis. “If approved, pegcetacoplan would be the first and only treatment for GA in Europe.”
“Pegcetacoplan is the only GA treatment to demonstrate increased effects over 24 months across a broad patient population,” said Frank G. Holz, DERBY and OAKS study investigator and professor and chairman of the department of ophthalmology at the University of Bonn, Germany. “There are currently no treatments to slow the progression of GA, a life-altering disease that causes substantial vision loss, so the potential approval of pegcetacoplan would be a significant milestone for patients in Europe.”
The MAA submission is based on results from the Phase 3 DERBY and OAKS studies at 24 months. In the studies, treatment with both every-other-month and monthly pegcetacoplan demonstrated robust and clinically meaningful reductions of GA lesion growth, with treatment effects that increased over time, and a favorable safety profile.
The U.S. marketing application is under review with a Prescription Drug User Fee Act (PDUFA) target action date of February 26, 2023.
About DERBY and OAKS
DERBY (n=621) and OAKS (n=637) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of pegcetacoplan with sham injections across a broad and representative population of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary objective of the studies is to evaluate the efficacy of pegcetacoplan in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence (p-value less than .05) at 12 months. Patients continued to receive masked treatment for 24 months.
About Geographic Atrophy (GA)
Geographic atrophy (GA) is an advanced form of age-related macular degeneration (AMD) and a leading cause of blindness that impacts more than five million people worldwide, including one million people in the United States.1,2 This progressive disease can severely impair visual function, independence, and quality of life as it takes on average 2.5 years for GA lesions to encroach the fovea, which is responsible for central vision.3 GA is caused by destruction of retinal cells through irreversible lesion growth that is driven by excessive complement activation.4 C3 is the only target that can precisely control the complement cascade due to its central location. There are currently no approved treatments for GA.
About Pegcetacoplan for Geographic Atrophy (GA)
Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy.
About Apellis
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in complement, we ushered in the first new class of complement medicine in 15 years with the approval of the first and only targeted C3 therapy. We are advancing this science to continually develop transformative medicines for people living with rare, retinal, and neurological diseases. For more information, please visit http://apellis.com or follow us on Twitter and LinkedIn.
1 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta analysis. Ophthalmology 2012;119:571–580.
2 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116.
3 Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.
4 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261.
SOURCE: Apellis
Post Views: 58
