Clinical Trial News

FDA approves Epysqli (eculizumab-aagh), a biosimilar to Soliris for treating paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), based on evidence showing high similarity to Soliris without clinically meaningful differences in safety, purity, and potency.

FDA approves Zituvimet XR, a sitagliptin and metformin hydrochloride extended-release tablet, for adults with type 2 diabetes, manufactured by Zydus Lifesciences Limited.

Phathom Pharmaceuticals announces FDA approval of Voquezna (vonoprazan) tablets for treating heartburn associated with Non-Erosive GERD in adults, marking a significant milestone for millions of GERD patients.

The FDA accepted Atara Biotherapeutics' BLA for tabelecleucel (tab-cel®) for EBV+ PTLD, granting Priority Review with a PDUFA date of January 15, 2025. Tab-cel is an allogeneic EBV-specific T-cell immunotherapy with Breakthrough Therapy and orphan drug designations, aiming to treat patients with at least one prior therapy. The BLA is supported by pivotal data from over 430 patients, showing a 48.8% Objective Response Rate. Atara has a global partnership with Pierre Fabre Laboratories for tab-cel commercialization, with potential milestone payments upon FDA approval.

Orexo received a complete response letter from the FDA for OX124, a high-dose naloxone rescue medication for opioid overdose, requiring an additional Human Factors study and technical data on the final commercial product. Orexo plans to address these requests and resubmit the NDA.

Lexicon Pharmaceuticals announced the FDA acknowledged the resubmission of its NDA for Zynquista (sotagliflozin) for type 1 diabetes and CKD, with a PDUFA goal date of December 20, 2024.

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.