Welcome to The Weekly Healthcare Update
This is your source for important healthcare news and advancements, tailored for busy healthcare professionals. Each week, we bring you five crucial updates from the healthcare sector, including new treatments, regulatory changes, and other innovations that could impact the field.
Here’s a summary of this week’s important stories, highlighting key actions from the US Food and Drug Administration (FDA) related to nephrology and ophthalmology. Notable developments include a Priority Review for pegcetacoplan related to rare kidney diseases, new approval for IgA nephropathy (IgAN), a Complete Response Letter (CRL) for reproxalap in treating dry eye disease (DED), and the first-ever therapy for IgG4-related disease. Additionally, the development of sozinibercept for wet age-related macular degeneration (AMD) has been halted.
Let’s take a closer look at these stories from the week of March 30, 2025.
FDA Prioritizes Pegcetacoplan (Empaveli) for C3G and IC-MPGN
The FDA has accepted Apellis Pharmaceuticals’ supplemental New Drug Application (sNDA) for pegcetacoplan (Empaveli), focusing on C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). This application is backed by encouraging results from a 26-week Phase 3 VALIANT trial, with a target decision date set for July 28, 2025.
Opthea Halts Sozinibercept Trials
Opthea has announced the termination of the ShORe and COAST trials for sozinibercept, a new treatment for wet AMD. The decision was made after the drug did not show the necessary vision improvement outcomes in the Phase 3 COAST trial.
Atrasentan (Vanrafia) Gets Accelerated Approval for IgAN
The FDA has granted accelerated approval to Novartis’ atrasentan (Vanrafia) for treating adults with primary IgA nephropathy (IgAN) who are at risk of rapid disease progression. This marks a significant addition to the available therapies, as it is the first selective endothelin A receptor antagonist approved for this condition.
Complete Response Letter Issued for Reproxalap
The FDA has issued a Complete Response Letter (CRL) concerning the New Drug Application (NDA) for reproxalap, a treatment for dry eye disease (DED). The FDA noted that the treatment did not show sufficient efficacy in controlled studies. Aldeyra Therapeutics plans to discuss the next steps with the FDA and hopes to resubmit the NDA by mid-2025, anticipating a six-month review period.
First IgG4-RD Treatment Approved: Inebilizumab (Uplizna)
In a significant advancement, the FDA has approved Amgen’s inebilizumab-cdon (Uplizna) as the first treatment for adults diagnosed with immunoglobulin G4-related disease (IgG4-RD). This approval is based on the positive outcomes from the MITIGATE trial, where the treatment demonstrated an 87% reduction in IgG4-RD flare-ups compared to a placebo over a 52-week period.
Stay tuned for more updates and insights to keep you informed about the latest in healthcare.
