Product: ANDEMBRY®

Company: CSL

About the Product:

ANDEMBRY® (garadacimab) is a first-in-class monoclonal antibody that redefines hereditary angioedema (HAE) treatment by targeting activated Factor XII, an upstream initiator of bradykinin-mediated swelling. By intervening at the top of the disease pathway, ANDEMBRY® is designed to deliver early, durable, and sustained protection from unpredictable, potentially life-threatening, spontaneous attacks of swelling.

Discovered and developed entirely by CSL, ANDEMBRY® fuses leading scientific experience with deep patient insights plus state-of-the-art formulation and device development, all leading to a once-monthly, small-volume dose delivered via a convenient autoinjector. It is remarkably well tolerated, safe, and leads to strong, durable efficacy. This combination of features marks a significant advance in HAE care and allows patients to lead fuller lives. It also demonstrates the power of CSL’s scientific persistence, its focus on enduring patient impact, and its willingness to disrupt itself in the service of bettering patients’ lives.

Product: AVLAYAH™ (tividenofusp alfa-eknm)

Company: Denali Therapeutics Inc.

About the Product:

AVLAYAH™, the first FDA-approved biologic designed to cross the blood-brain barrier (BBB), represents a breakthrough for individuals with a debilitating rare disease and a pioneering innovation for brain delivery engineering. Enabled by Denali’s TransportVehicle™, AVLAYAH is the first enzyme replacement therapy designed to reach the brain and body, receiving FDA approval for treatment of neurologic manifestations of Hunter syndrome when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment. AVLAYAH clinically validates the TransportVehicle and establishes proof-of-concept that biologics can be engineered to overcome the BBB, with broad potential across neurodegenerative diseases.

Product: LYFGENIA™

Company: Genetix Biotherapeutics Inc.

About the Product:

LYFGENIA (lovotibeglogene autotemcel) is a one-time ex vivo gene addition cell therapy, FDA approved in December 2023 for patients 12 and older with sickle cell disease who have a history of vaso-occlusive events. As a one-time transformative treatment, LYFGENIA is made specifically for each patient, using the patient’s own blood stem cells. It adds functional copies of the beta-globin gene to a patient’s cells leading to production of anti-sickling hemoglobin that has shown to decrease or stop vaso-occlusive events.

Product: TALVEY® (talquetamab-tgvs)

Company: Johnson & Johnson

About the Product:

TALVEY® is the first and only approved GPRC5D-directed CD3 T-cell engager for adults with relapsed or refractory multiple myeloma after at least four prior therapies. It binds CD3 on T-cells and GPRC5D on myeloma cells, redirecting T-cells to attack the cancer. Targeting this novel antigen expands immunotherapy options and has demonstrated deep, durable responses in heavily pretreated patients. In the Phase 3 MonumenTAL-6 study, TALVEY®-based combinations significantly improved progression-free and overall survival versus standard care in patients after one to four prior lines of therapy, supporting TALVEY®’s potential in earlier treatment and redefining the multiple myeloma treatment landscape.

Product: ABRYSVO® oxygen mask

Company: Pfizer Inc.

About the Product:

A vaccine to help prevent respiratory syncytial virus (RSV) disease had been an elusive public health goal for more than half a century. Pfizer’s ABRYSVO® is the only RSV vaccine approved to help protect two distinct populations: individuals 18 years of age and older, as well as infants from birth through six months through maternal immunization. ABRYSVO is a bivalent vaccine that help provide protection against lower respiratory tract disease caused by the respiratory syncytial virus, inclusive of both major subgroups RSV-A and RSV-B. ABRYSVO continues Pfizer’s commitment to helping prevent serious respiratory disease in infants and older adults.

Product: PADCEV®

Company: Pfizer Inc./Astellas

About the Product:

PADCEV® (enfortumab vedotin), a first-in-class Nectin-4–directed antibody-drug conjugate has redefined bladder cancer treatment delivering the first major treatment advance in decades. In combination with pembrolizumab, PADCEV nearly doubled overall survival versus platinum-based chemotherapy in locally advanced or metastatic urothelial cancer—irrespective of cisplatin eligibility—displacing platinum as the first-line standard. This paradigm shift now extends into muscle-invasive disease, where perioperative PADCEV-based therapy has significantly improved event-free and overall survival in curative-intent care. Pfizer is committed to ongoing research exploring novel combinations and bladder preservation, offering the prospect of cure without life-altering surgery—a generational transformation in patient outcomes.

Product: IMAAVY®

Company: Johnson & Johnson

About the Product:

Nipocalimab (IMAAVY®) is a fully human, aglycosylated IgG1 monoclonal antibody that selectively blocks the neonatal Fc receptor (FcRn), accelerating clearance of pathogenic IgG autoantibodies while preserving non-IgG immune functions. Nipocalimab is the first FcRn blocker FDA-approved by April 2025 for both anti-AChR– and anti-MuSK–positive generalized myasthenia gravis in adults and adolescents aged 12 and older. Administered as a consistent dosing regimen, nipocalimab delivers rapid, sustained IgG reduction without broad immunosuppression, addressing a critical unmet need across ages and serotypes in gMG, and establishing an innovative platform for treatment across multiple IgG-mediated autoimmune/alloimmune diseases.