EU Approves CSL and Arcturus' KOSTAIVE®, First Self-amplifying mRNA COVID-19 Vaccine

CSL, a leader in global biotechnology, and Arcturus Therapeutics, a pioneer in self-amplifying mRNA technology, have reached a significant milestone with the European Commission (EC) granting marketing authorization for their COVID-19 vaccine, KOSTAIVE. This vaccine is designed for individuals aged 18 and older and marks the first approval of its kind for a self-amplifying mRNA COVID-19 vaccine by the EC. KOSTAIVE is already available in Japan to combat COVID-19.

The decision by the European Commission follows a positive assessment from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) made in December 2024. The approval enables centralized marketing authorization, extending its validity to all European Union (EU) member states as well as countries in the European Economic Area (EEA).

Jonathan Edelman, MD, Senior Vice President of the Vaccines Innovation Unit at CSL, expressed that the EC's approval is a pivotal achievement in their ongoing efforts to advance KOSTAIVE. The company is committed to enhancing the vaccine's formulation to better serve healthcare providers and their patients. As COVID-19 continues to pose a global threat, CSL is focused on finalizing technical improvements and making this innovative vaccine widely accessible across Europe.

The vaccine's approval is grounded in robust clinical data obtained from several studies, including an extensive phase 1/2/3 study that highlighted the vaccine’s efficacy and tolerability. Phase 3 trials for COVID-19 boosters revealed that KOSTAIVE achieved superior immunogenicity compared to traditional mRNA COVID-19 vaccines. Furthermore, follow-up analysis of a booster dose demonstrated that KOSTAIVE offers enhanced immunogenicity and sustained antibody presence for up to 12 months post-vaccination, effectively targeting various SARS-CoV-2 variants across different age groups.

Joseph Payne, CEO of Arcturus, emphasized the transformative potential of KOSTAIVE and its underlying sa-mRNA technology. The approval underscores the clinical promise of the vaccine, providing a pathway for more comprehensive and enduring protection against the constantly evolving COVID-19 virus.

Self-amplifying mRNA vaccines represent a leap forward in vaccine development, offering a mechanism where the body is instructed to produce more mRNA and protein, thereby amplifying the immune response. This contrasts with standard mRNA vaccines, which do not have this self-amplifying feature.

CSL, with over a century of experience, is renowned for its portfolio of lifesaving medicines, including therapies for hemophilia, immune deficiencies, vaccines for influenza, and treatments for iron deficiency and nephrology. CSL's global presence spans over 100 countries, backed by its 32,000 employees dedicated to delivering innovative healthcare solutions.

Arcturus, founded in 2013 and headquartered in San Diego, focuses on the development of commercial mRNA medicines and vaccines. The company utilizes advanced technologies such as LUNAR lipid-mediated delivery and STARR mRNA technology. Arcturus is notable for its development of KOSTAIVE, the first self-amplifying mRNA COVID-19 vaccine approved globally. Their collaboration with CSL Seqirus extends to a joint venture in Japan aimed at producing mRNA vaccines and therapeutics. Arcturus continues to diversify its pipeline with RNA therapeutic candidates addressing conditions such as ornithine transcarbamylase deficiency and cystic fibrosis, alongside its partnership-driven mRNA vaccine programs targeting COVID-19 and influenza.

The recent European Commission approval of KOSTAIVE underscores the potential of self-amplifying mRNA vaccines in the fight against infectious diseases, marking a significant step forward in protecting global populations from the persistent threat of COVID-19.