New products to market CSL continues to broaden the geography and use of our medicines for rare and specialty diseases across the globe within CSL’s immunology, haematology, nephrology and transplant therapeutic areas as well as in iron deficiencies, and the use of vaccines to help prevent infectious disease and protect public health. Within the immunology portfolio, regulatory indication expansion and new registrations are primarily focused on CSL’s subcutaneous immunoglobulin HIZENTRA® and human C1-esterase inhibitor BERINERT®. There was one new HIZENTRA® registration for primary immunodeficiency (PID), a chronic disorder in which part of the body’s immune system is missing or malfunctioning. Indication expansion was approved for HIZENTRA® for secondary immunodeficiency (SID) in six countries and chronic inflammatory demyelinating polyneuropathy (CIDP) in one country. SID is similar to primary immunodeficiency (PID); however SID occurs when the immune system is compromised as a result of disease or due to an environmental factor (e.g., chemotherapy, disease complication). CIDP is a chronically progressive, rare autoimmune disorder that affects the peripheral nerves and may cause permanent nerve damage. With CIDP, the myelin sheath, or the protective covering of the nerves, is damaged, which may result in numbness or tingling, muscle weakness, fatigue and other symptoms, which worsen over time. In addition, there were new registrations in two countries each for BERIRAB® (human rabies immunoglobulin) and BERINERT® and new registrations in one country each for HAEGARDA®, PRIVIGEN®, TETAGAM® and HEPATITIS B Immunoglobulin P Behring. HEMGENIX® (etranacogene dezaparvovec) is the first, one-time gene therapy treatment for adults with haemophilia B. In October 2023, CSL and uniQure were awarded the prestigious 2023 Prix Galien USA Award for HEMGENIX® in the category of Best Product for Rare/Orphan diseases and was a finalist for the 2024 International Prix Galien Award as “Best of the Best” new Rare/Orphan Diseases category. The Prix Galien USA is America’s preeminent prize acknowledging the leading-edge of scientific advances in life sciences. The biennial awards are presented by The Galien Foundation, the premier global institution dedicated to honouring innovators in life sciences and bringing together life science innovators around the world. Therapeutic Areas and Product Portfolio Health authorities and national immunisation technical advisory groups (NITAGS) worldwide have recommended removing B/Yamagata lineage viruses from seasonal influenza vaccines following WHO confirmation of their undetectable circulation. In the United States, the FDA has approved the transition of CSL’s seasonal influenza vaccines – FLUAD®, FLUCELVAX® and AFLURIA® – from quadrivalent to trivalent formulations for the upcoming Northern Hemisphere 2024/2025 influenza season. CSL continues to work closely with global health authorities to transition to trivalent seasonal influenza vaccines in each country as quickly as local regulatory pathways and timelines allow. Within the iron deficiency portfolio, there were new product registrations in three countries and two label expansions for FERINJECT® (ferric carboxymaltose) for the treatment of iron deficiency. KOSTAIVE®, CSL’s sa-mRNA COVID-19 vaccine, has demonstrated a stronger, broader, and more durable immune response compared to an approved conventional mRNA vaccine. These attributes address significant unmet medical needs in the COVID-19 vaccine market. In December 2023, KOSTAIVE® was approved for use in Japan for individuals 18 years and older, marking it as the first ever approved sa-mRNA vaccine. Submission for approval in Europe is in progress, with plans for future filings globally. In CSL’s haematology therapeutic area, there is a continued focus on the expansion of the current portfolio as well as further registrations of HEMGENIX®, etranacogene dezaparvovec, a one‑time gene therapy for the treatment of adults with haemophilia B with three new registrations. New registrations were achieved in seven countries for IDELVION®, CSL’s recombinant factor IX albumin fusion protein (rFIX-FP) which is used to control and prevent bleeding episodes in people with haemophilia B. New product registrations were achieved in three countries for AFSTYLA®, CSL’s recombinant factor VIII which is used to control and prevent bleeding episodes in people with haemophilia A. New product registrations were achieved for CSL’s human coagulation factor VIII/vWF HAEMATE® in three countries. One new product registration was achieved for each of BERIPLEX®, CSL’s human prothrombin complex concentrate, and HAEMOCOMPLETTAN® P, CSL’s human fibrinogen concentrate. In Nephrology and Transplant, first time approval was received in the European Union for FILSPARI®, CSL’s dual endothelin A and angiotensin 1 receptor antagonist for the treatment of adults with primary immunoglobulin A nephropathy (IgAN). There were four new product registrations for TAVNEOS® (avacopan) for the treatment of adults with severe active anti‑neutrophil cytoplasmic autoantibody (ANCA)‑associated vasculitis. There was one new product registration for KORSUVA® (difelikefalin) for the treatment of moderate-to‑severe pruritus associated with chronic kidney disease in adult patients on haemodialysis, and two new indication expansions for VELTASSA® (patiromer sorbitex calcium) for the treatment of high blood potassium. For Vaccines, indication expansion was the focus for the seasonal influenza portfolio with new patient populations approved in four markets and one new registration for each of FLUAD® QUADRIVALENT, CSL’s adjuvanted influenza vaccine, and CELLDEMIC®, CSL’s influenza vaccine to protect against the H5N1 subtype of the influenza A virus (sometimes called ‘bird flu’ or ‘avian flu’). Additionally, there was one new registration for each of CSL’s pandemic influenza vaccines, INCELLIPAN®, PANVAX® and FOCLIVIA® Influenza A (H5N1) and two indication expansions were approved for FOCLIVIA®. 24 Limited Annual Report 2023/24
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