VERTEX PHARMACEUTICALS INC / MA

B USINESS OVERVIEW We are a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases, with a focus on specialty markets. We have approved medicines for cystic fibrosis ( CF ), sickle cell disease ( SCD ), transfusion dependent beta thalassemia ( TDT ), and acute pain, and we continue to serially innovate and advance next-generation clinical and research programs in these areas. O ur mid- and late-stage clinical pipeline includes programs across a range of modalities in additional serious diseases, including IgA nephropathy, APOL1-mediated kidney disease, neuropathic pain, type 1 diabetes, primary membranous nephropathy, autosomal dominant polycystic kidney disease, and myotonic dystrophy type 1 . The following chart sets forth our approved products, clinical-stage programs, and select pre-clinical programs: We are advancing five pivotal programs across multiple disease areas: IgA Nephropathy . We are developing povetacicept, a dual inhibitor of the B cell activating factor ( BAFF ) and a proliferation-inducing ligand ( APRIL ) pathways, as a potentially best-in-class approach to treat IgA nephropathy ( IgAN ), a serious, progressive, life-threatening kidney disease that often progresses to end-stage renal disease . We completed enrollment in the IgAN Phase 3 clinical trial and submitted the first module of the rolling Biologics Licensing Application ( BLA ) for povetacicept in IgAN in the fourth quarter of 2025. We expect to complete the submission for potential accelerated approval in the U.S.