Csl Ltd Stock Price, News & Analysis

CSL Vifor and Travere Therapeutics have achieved a significant milestone as the European Commission converts the conditional approval of FILSPARI to standard marketing authorization for treating IgA Nephropathy (IgAN).

FILSPARI, the only Dual Endothelin Angiotensin Receptor Antagonist (DEARA) approved in Europe for IgAN treatment, is currently available in Germany, Austria, and Switzerland. The approval covers all EU member states, Iceland, Liechtenstein, and Norway.

The decision follows positive phase-III PROTECT study results, which showed FILSPARI significantly slowed kidney function decline over two years compared to irbesartan. The drug is approved for adults with primary IgA nephropathy with specific urine protein excretion levels.

This standard approval, granted without changes to the indication, validates the clinical data and marks an important advancement for IgAN patients across Europe.

CSL Behring has received approval from Japan's MHLW for ANDEMBRY® (garadacimab), a first-in-class monoclonal antibody treatment for preventing hereditary angioedema (HAE) attacks. The treatment, administered via pre-filled pen for once-monthly subcutaneous injection, works by inhibiting activated Factor XII, the initiating factor in the HAE pathway.

The approval follows successful results from the international Phase 3 VANGUARD trial, with the detailed results published in The Lancet (April 2023) and Allergy (October 2024). ANDEMBRY has already received approvals in Australia, the United Kingdom, and the European Union.

HAE is a rare genetic disorder affecting approximately 430 diagnosed patients in Japan, with an estimated total patient population of 2,500 based on global prevalence rates of 1 in 50,000 people. The condition causes recurrent and unpredictable attacks of angioedema in multiple body sites.

CSL (CSLLY) and Arcturus Therapeutics announced that the European Commission has granted marketing authorization for KOSTAIVE®, the first self-amplifying mRNA COVID-19 vaccine, for individuals 18 years and older. The approval follows a positive CHMP opinion from December 12, 2024, and is valid across all EU member states and EEA countries.

KOSTAIVE, already marketed in Japan, demonstrated superior immunogenicity and antibody persistence for up to 12 months post-vaccination compared to conventional mRNA COVID-19 vaccines in clinical trials. The approval is based on positive data from multiple studies, including an integrated phase 1/2/3 study and Phase 3 booster trials, which showed higher immunogenicity results versus a conventional mRNA COVID-19 vaccine comparator.

CSL and Arcturus Therapeutics (ARCT) have received European Commission marketing authorization for KOSTAIVE®, the first self-amplifying mRNA (sa-mRNA) COVID-19 vaccine for individuals 18 and older. The approval follows a positive CHMP opinion from the European Medicines Agency on December 12, 2024.

KOSTAIVE demonstrated superior immunogenicity and antibody persistence for up to 12 months post-vaccination compared to conventional mRNA COVID-19 vaccines in clinical trials. The approval is based on data from multiple studies, including an integrated phase 1/2/3 study showing efficacy and tolerability, and Phase 3 booster trials.

The vaccine is already marketed in Japan and will be valid across all EU member states and EEA countries. CSL is working to optimize KOSTAIVE's formulation to better serve healthcare professionals and patients in Europe.

CSL (CSLLY) has received European Commission approval for ANDEMBRY® (garadacimab), the first and only once-monthly treatment targeting factor XIIa for preventing hereditary angioedema (HAE) attacks in patients aged 12 and older. This marks the third regulatory approval for ANDEMBRY, following recent approvals in Australia and the UK.

The treatment comes with a patient-centric pre-filled pen for subcutaneous self-injection and works by inhibiting plasma protein factor XIIa. The approval is based on the Phase 3 VANGUARD trial and its open-label extension study, with results published in The Lancet (April 2023) and Allergy (October 2024).

HAE is a rare genetic disorder affecting approximately 1 in 50,000 people, characterized by recurrent and unpredictable attacks of angioedema. The treatment will be available following access and reimbursement negotiations, and is currently under review in the US, Japan, Switzerland, and Canada.

CSL Behring announced four-year results from the pivotal HOPE-B study of HEMGENIX, their gene therapy for hemophilia B. The data demonstrates sustained efficacy and safety through four years post-treatment. Key findings include:

- 94% of patients remained free of continuous factor IX prophylaxis
- Mean factor IX activity levels maintained at 37% through four years
- Approximately 90% reduction in mean adjusted annualized bleeding rate from lead-in to year four
- Joint bleeds reduced from 2.34 to 0.09 mean ABR

The Phase III HOPE-B trial involved 54 adult male participants with severe or moderately severe hemophilia B. No serious adverse events related to HEMGENIX were reported, with 96% of treatment-related adverse events occurring in the first six months. HEMGENIX, approved by FDA in 2022, is the first gene therapy for hemophilia B that can treat patients with and without AAV5 neutralizing antibodies.

CSL (ASX:CSL; USOTC:CSLLY) received a positive CHMP opinion recommending marketing authorization for garadacimab, a first-of-its-kind once-monthly treatment for hereditary angioedema (HAE) prevention in patients aged 12 and older. The treatment, which targets factor XIIa to prevent HAE attacks, demonstrated impressive results in the Phase 3 VANGUARD trial, with 62% of patients achieving attack-free status and an 86.5% reduction in mean monthly HAE attacks compared to placebo.

The European Commission's final decision is expected in Q1 2025. The recommendation is supported by favorable long-term safety data from an ongoing open-label extension study, with median exposure of 13.8 months. If approved, garadacimab would be available across all EU member states.

CSL Behring, a global leader in biotherapeutics, has significantly scaled its healthcare professional (HCP) engagement program using the ON24 platform. Leveraging ON24's intelligent engagement solutions, CSL Behring has enhanced its digital engagement, resulting in new potential customers and better insights into customer content engagement. The integration with Veeva CRM allows real-time data capture and analysis, informing follow-up strategies and deepening customer relationships. Before partnering with ON24, CSL Behring faced challenges with disjointed digital tools and manual processes that hindered scalability. Now, the company offers consistent, meaningful content to HCPs globally through live and on-demand webinars, reaching thousands of HCPs and increasing engagement. This transformation has enabled CSL Behring to expand its reach without adding headcount, repurposing content across 40 countries. The life sciences industry saw a 13% year-over-year increase in digital experiences in 2023, emphasizing the sector's shift towards omnichannel strategies.