Clinical Trial News

Eton Pharmaceuticals' NDA for ET-400, a hydrocortisone oral solution, was accepted by the FDA with a PDUFA target action date of February 28, 2025.

Uncommon Cures, a U.S. company, launched a boutique clinical trial program for rare diseases, underestimating the demand. With eight contracts and talks with over 50 clients, CEO Marshall Summar highlights the need for a new model blending regulatory tasks with a concierge-based facility. The model, expanding internationally with sites in D.C., Utah, Chile, Switzerland, and UAE, aims to streamline trials, reduce costs by over 40%, and improve patient access globally.

FDA approves Arcutis' Zoryve (roflumilast) cream 0.15% for treating mild to moderate atopic dermatitis in adults and children from 6 years old, offering a once-daily, steroid-free option with rapid disease clearance and significant itch reduction.

FDA approves Genentech's Vabysmo Prefilled Syringe (PFS) for wet age-related macular degeneration (AMD), diabetic macular edema (DME), and macular edema following retinal vein occlusion (RVO), affecting nearly 3 million people in the U.S. Vabysmo PFS, a bispecific antibody, offers a ready-to-use format, enhancing treatment simplicity and effectiveness.

FDA approves Kisunla (donanemab-azbt) for early symptomatic Alzheimer's disease, targeting amyloid plaques. Kisunla, a once-monthly IV infusion, shows potential to stop therapy upon plaque removal, reducing costs and infusions. Clinical trials demonstrate significant slowing of clinical decline and amyloid plaque reduction, though risks include amyloid-related imaging abnormalities (ARIA) and infusion-related reactions.

FDA issued a draft guidance on Diversity Action Plans to improve enrollment of underrepresented populations in clinical studies, required under FDORA 2022. The plans aim to increase representation based on race, ethnicity, sex, and age, and must specify enrollment goals and strategies. Comments on the draft are due by September 26, 2024.

Vertex's vanzacaftor/tezacaftor/deutivacaftor triple therapy NDA accepted by FDA for cystic fibrosis, targeting those aged 6+ with at least one F508del or responsive mutation in CFTR gene. PDUFA action date set for January 2, 2025, with EMA validation also received.

FDA approves FYB203/Ahzantive® (aflibercept-mrbb), a biosimilar to Eylea®, for treating nAMD, DME, DR, and RVO, inhibiting VEGF. FYB203/Ahzantive® demonstrated comparable efficacy and safety to Eylea®, with global sales of around US$ 9 billion in 2023. A marketing authorization application for FYB203 was submitted to the EMA, with a decision expected by early 2025.

FDA approves Nypozi (filgrastim-txid), a biosimilar to Neupogen, indicated to decrease the incidence of infection in patients with non-myeloid malignancies.

SpringWorks Therapeutics completes NDA submission to FDA for mirdametinib, an investigational MEK inhibitor for treating NF1-PN in children and adults. The submission includes data from the Phase 2b ReNeu trial, showing significant response rates, deep and durable responses, and a manageable safety profile. Mirdametinib has received Orphan Drug and Fast Track designations from the FDA and is planned for MAA submission to the EMA in the second half of 2024.