The U.S. Food and Drug Administration (FDA) has released the 2025 Accelerating Rare disease Cures (ARC) Program Annual Report. Since its launch in 2022, the Center for Drug Evaluation and Research (CDER) ARC Program continues to be a key resource for the rare disease community and a driver of innovative approaches for rare disease drug development. …
FDA Releases 2025 CDER ARC Program Annual Report Read More »
Regeneron Pharmaceuticals and Tessera Therapeutics are collaborating to advance a promising in vivo gene editing therapy for alpha-1 antitrypsin deficiency (AATD).
Johnson & Johnson’s immunoselective monoclonal antibody Imaavy (nipocalimab) has been approved by the European Commission to treat individuals with generalised myasthenia gravis (gMG) 12 years old and over.
On December 3, 2025, the Food and Drug Administration granted traditional approval to pirtobrutinib (Jaypirca, Eli Lilly and Company) for adults with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have previously been treated with a covalent BTK inhibitor. In 2023, FDA granted accelerated approval to pirtobrutinib for adults with CLL/SLL who have …
Otsuka Receives FDA Accelerated Approval for VOYXACT® (sibeprenlimab-szsi) for the Reduction of Proteinuria in Adults with Primary Immunoglobulin A Nephropathy (IgAN) at Risk for Disease Progression
