FDA expands approval of Tzield to youngest type 1 diabetes patients

The US Food and Drug Administration (FDA) has expanded approval of Sanofi’s teplizumab (Tzield) to include children as young as one year old with stage 2 type 1 diabetes, marking a significant extension of the first disease-modifying therapy for the condition. The decision allows earlier intervention to delay progression to stage 3 disease, when patients typically require lifelong insulin therapy.

The approval is supported by Phase 4 PETITE-T1D data evaluating safety and pharmacokinetics in young children, with the therapy administered as a 14-day intravenous course. By targeting CD3 on T cells, teplizumab aims to modulate the autoimmune response responsible for beta-cell destruction, potentially preserving insulin production. Source

FDA signals shift away from animal testing in drug development

The FDA has announced plans to phase out animal testing requirements for monoclonal antibodies and other drugs, marking a significant shift toward human-relevant testing methods. The new roadmap encourages the use of New Approach Methodologies (NAMs), including AI-based computational models, organoids, and organ-on-a-chip systems, to assess drug safety and predict toxicity.

The initiative will begin immediately for investigational new drug applications, with regulators also considering real-world human data from international studies where appropriate. The FDA says the approach could improve safety prediction, accelerate development timelines, and reduce costs, while minimizing reliance on animal models. A pilot program is planned to support developers using primarily non-animal testing strategies, with broader regulatory updates expected as these methods are validated. Source

IL-33 biologic reduces exacerbations in Phase III COPD trial

AstraZeneca has reported positive top-line results from the Phase III MIRANDA trial showing that its IL-33-targeting antibody, tozorakimab, significantly reduced moderate-to-severe exacerbations in patients with chronic obstructive pulmonary disease (COPD). The study met its primary endpoint in both the former smoker population and the overall cohort, which included patients across all eosinophil levels and disease severities, highlighting the therapy’s broad potential applicability.

Patients receiving tozorakimab on top of standard inhaled therapy experienced clinically meaningful reductions in exacerbation rates, with the treatment generally well tolerated and consistent with previous studies. The results add to earlier positive Phase III data from the OBERON and TITANIA trials, strengthening the case for tozorakimab as a potential first-in-class biologic targeting IL-33. By inhibiting both reduced and oxidized forms of the cytokine, the therapy aims to address upstream drivers of inflammation and mucus dysfunction in COPD, a disease where many patients continue to experience exacerbations despite standard care. Source

Dupixent approved in EU for young children with chronic hives

The European Commission has approved dupilumab (Dupixent) for children aged 2 to 11 years with moderate-to-severe chronic spontaneous urticaria (CSU), marking the first targeted therapy for this population. The decision expands the drug’s use beyond adults and adolescents to younger patients who remain symptomatic despite antihistamine treatment and have limited therapeutic options.

The approval is based on data from the Phase III LIBERTY-CUPID program, including adult placebo-controlled trials and pediatric data from the CUPIDKids study. Dupixent significantly reduced itch and hive severity and increased rates of disease control compared with standard care alone. By inhibiting IL-4 and IL-13 signaling, the therapy targets key drivers of type 2 inflammation, extending its established mechanism across multiple chronic inflammatory diseases in younger patients. Source 

Biocon secures Health Canada approval for denosumab biosimilars

Biocon Biologics has received Health Canada approval for Bosaya and Vevzuo, biosimilars to denosumab reference products Prolia and Xgeva. The approvals expand the company’s oncology and bone health portfolio, covering indications related to osteoporosis and the prevention of skeletal-related events in patients with bone metastases.

Denosumab is a monoclonal antibody targeting RANKL, a key regulator of bone resorption. The biosimilars were evaluated against the reference products for similarity in efficacy, safety, and pharmacokinetics. Source

Moderna’s combination flu-COVID vaccine wins EU approval

The European Commission has granted marketing authorization to Moderna’s mCOMBRIAX, the first mRNA-based combination vaccine targeting both influenza and COVID-19. Approved for adults aged 50 and older, the vaccine combines protection against two major respiratory viruses in a single dose, aiming to simplify immunization strategies and improve uptake in higher-risk populations.

The approval is based on Phase III data showing that the vaccine met all primary endpoints for immune response, demonstrating non-inferiority to co-administered influenza and COVID-19 vaccines and, in many cases, higher immunogenicity against key influenza strains and SARS-CoV-2. The vaccine also showed an acceptable safety profile consistent with existing vaccines. Source

For this week’s cell and gene therapy news – including Lilly’s $7B Kelonia acquisition, AACR updates on next-generation CAR-T designs, and emerging clinical data across solid tumors and autoimmune disease – click here