Biohaven disappointed as rare disease therapy faces FDA setback 'despite promising results'

The FDA setback highlights ongoing tensions between patient needs, regulatory processes, and the use of real-world evidence in evaluating treatments for rare diseases

Sumitomo Pharma America unveils promising new leukemia and myelofibrosis data ahead of ASH 2025

Enzomenib, an oral, small-molecule inhibitor designed to disrupt the menin-MLL protein interaction involved in leukemogenesis

Aldeyra refocuses pipeline on next-gen RASP modulators despite positive phase II success

ADX-248, which replaces ADX-743, is being developed for the treatment of metabolic inflammation, including obesity and hypertriglyceridemia

Alexion highlights breakthrough C5 inhibition advances in gMG at 2025 AANEM and MGFA scientific sessions

New Phase III data on gefurulimab highlight potential as a self-administered treatment option; real-world evidence reinforces clinical benefits of Ultomiris and Soliris

FDA grants fast track status to Heidelberg Pharma’s Amanitin-based ADC candidate for multiple myeloma

The Fast Track Designation was supported by nonclinical data as well as clinical results from the ongoing Phase I/IIa trial evaluating the safety and tolerability

FDA approves Teva’s expanded indication for Uzedy for bipolar I disorder

UZEDY (risperidone) extended-release injectable suspension for subcutaneous use is indicated for use every one or two months for the treatment of schizophrenia in adults

ACL Digital acquires US-based CRO Symbiance to expand clinical research capabilities

The acquisition positions ACL Digital as an end-to-end provider of global clinical research and data CRO solutions

Biocon Biologics secures US market entry date for Bosaya and Aukelso, Denosumab biosimilars

Moderna’s mNEXSPIKE generates strong immune response against LP.8.1 variant in humans

Both of Moderna's COVID-19 vaccines for the 2025-2026 season have demonstrated strong immune responses against today's top circulating COVID-19 strains

Ascletis reports positive phase Ib results for oral GLP-1R agonist ASC30 at EASD 2025

The Phase Ib MAD study is a randomized, double-blind, placebo-controlled trial conducted in the United States