APELLIS PHARMACEUTICALS, INC

EMA Validates Aspaveli® Extension Application for Rare Kidney Diseases C3G and IC-MPGN

3 months ago

• The European Medicines Agency has validated the application to extend Aspaveli® (pegcetacoplan) use for treating C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis, two rare kidney diseases currently lacking approved treatments. • Phase 3 VALIANT study demonstrated significant 68% reduction in proteinuria with pegcetacoplan compared to placebo (p<0.0001), along with stabilization of kidney function and reduced C3c staining intensity. • The potential approval could provide the first targeted treatment for approximately 8,000 European patients affected by these conditions, with possible US launch in second half of 2025.

Apellis' Syfovre Approved in Australia for Geographic Atrophy

4 months ago

• Apellis Pharmaceuticals' Syfovre (pegcetacoplan) has been approved in Australia for treating geographic atrophy (GA) secondary to age-related macular degeneration (AMD). • Syfovre is the first and only approved treatment for GA in Australia, offering a new option to slow the progression of this irreversible vision loss. • The approval is based on Phase 3 OAKS and DERBY studies, which demonstrated that Syfovre slowed GA progression with a generally well-tolerated safety profile. • Affecting over 75,000 Australians, GA leads to progressive vision loss, impacting independence and quality of life, making this approval a significant advancement.

FDA Approves Expanded Label for Astellas' Izervay, Removing Dosing Duration Limits for Geographic Atrophy

4 months ago

• The FDA approved an expanded label for Astellas' Izervay, removing the limitation on dosing duration for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). • The approval was based on positive two-year results from the GATHER2 Phase 3 clinical trial, demonstrating the efficacy and safety of Izervay with extended use. • The GATHER2 study showed that Izervay continued to reduce the rate of GA lesion growth over two years, with benefits observed as early as 6 months. • With over 210,000 vials distributed by the end of December 2024, post-marketing safety data remains consistent with clinical trials, reinforcing prescriber confidence.

Pegcetacoplan Shows Strong Efficacy in C3G and IC-MPGN Patients: VALIANT Trial Results

7 months ago

• Pegcetacoplan demonstrated a statistically significant 68% reduction in proteinuria compared to placebo in patients with C3G and IC-MPGN. • The VALIANT trial showed pegcetacoplan stabilized eGFR, a key measure of kidney function, with a significant difference compared to placebo. • A substantial proportion of patients treated with pegcetacoplan achieved complete clearance of C3c deposits, a key marker of disease activity. • Pegcetacoplan exhibited a favorable safety profile, with similar rates of adverse events compared to the placebo group in the VALIANT trial.

Pegcetacoplan Shows Strong Results in VALIANT Trial for C3G and IC-MPGN

7 months ago

• Pegcetacoplan significantly reduced proteinuria by 68% compared to placebo in patients with C3G and IC-MPGN, with improvements seen as early as Week 4. • The VALIANT study demonstrated stabilization of eGFR and a substantial reduction in C3c staining intensity in patients treated with pegcetacoplan. • Pegcetacoplan exhibited a favorable safety profile and high compliance, with similar rates of adverse events compared to the placebo group. • Apellis plans to submit regulatory applications to the FDA and EMA for pegcetacoplan as a treatment for C3G and IC-MPGN in early 2025.

Pegcetacoplan Shows Significant Treatment Effect in C3G and IC-MPGN Patients

7 months ago

• Pegcetacoplan significantly reduced proteinuria by 68% compared to placebo in patients with C3G and primary IC-MPGN, with improvements seen as early as Week 4. • The VALIANT study demonstrated that pegcetacoplan stabilized eGFR, a key measure of kidney function, and substantially reduced C3c staining intensity. • All secondary endpoints favored pegcetacoplan, including composite renal endpoint and proteinuria reduction, with a favorable safety profile observed. • Sobi plans to submit a marketing application to the EMA in 2025, while Apellis intends to submit to the FDA in early 2025.

Pegcetacoplan Shows Promise in Treating Rare Kidney Diseases C3G and IC-MPGN

7 months ago

• Phase 3 VALIANT study reveals pegcetacoplan significantly reduces proteinuria by 68% in patients with C3G and IC-MPGN compared to placebo. • Pegcetacoplan stabilizes eGFR, a key measure of kidney function, and substantially reduces C3c deposits, a marker of disease activity. • The treatment demonstrates a favorable safety profile, with adverse event rates similar between the pegcetacoplan and placebo groups. • Apellis plans to submit regulatory applications to the FDA and EMA for pegcetacoplan as a treatment for C3G and IC-MPGN in early 2025.