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Australia and New Zealand LRA Watchdog Update - June 2025

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Paula Stoddart
Clinical Manager, Miltenyi Biotec Australia

Jessica Sue
Quality Manager, Sydney Cord Blood Bank, Sydney Children’s Hospitals Network

AUSTRALIAN AND NEW ZEALAND REGULATORY UPDATES

AOTEAROA – NEW ZEALAND

Medsafe

On 31^st March 2025 the NZ Medicines Amendment Bill was introduced to the House of Representatives. The Medicines Amendment Bill amends the Medicines Act 1981 to improve access to medicines. The Bill does this by:

introducing a streamlined verification pathway for medicines approval in New Zealand, and;
updating prescribing settings to enable wider prescribing of unapproved medicines in appropriate circumstances.

The Bill also updates details of the membership and terms of the Medicines Classification Committee in the Medicines Act. The Bill has passed its first reading and has been referred to the Health Select Committee for consideration^[¹^].

AUSTRALIA

Therapeutic Goods Administration (TGA)

The new Procedure for Recalls, Product Alerts and Product Corrections (PRAC) took effect on 5^th March 2025.

The PRAC replaces the Uniform Recall Procedure for Therapeutic Goods (URPTG) as the procedure for sponsors to follow when conducting market actions in Australia

The PRAC will:

enhance the transparency, communication and timeliness of recalls, alerts and corrections;
introduce new market action terminology, reducing the categories from 8 to 4;
halve the number of steps in the process from 10 to 5 but will not require any major changes to how sponsors approach the process;
feature fewer pages with less repetition, clearer information and instructions.

One of the most requested changes was to update the recall terminology, removing the sometimes confusing categories of ‘recall’ and ‘non-recall’ actions, and replacing them with one category called ‘market actions’^[²^,³^].

The TGA released version 1.0 of the Good Clinical Practice Inspection Program (GCPIP) 2023-2024 in March 2025. The GCPIP inspects trials of medicines and biologicals and commenced in 2022, with inspectors assessing whether Australian clinical trials sites are meeting their GCP responsibilities, to safeguard the safety and wellbeing of participants. The report provides a summary of GCPIP activities throughout from 1^st January 2023 to 31^st December 2024, including where there was both compliance and non-compliance to GCP activities and examples of critical and major deficiencies. The report can be read here: https://www.tga.gov.au/sites/default/files/2025-04/good-clinical-practice-inspection-program-2023-2024-250407.pdf.

Office of the Gene Technology Regulator (OGTR)

The OGTR has granted a license (DIR 210, issued April 2025) that allows Doherty Clinical Trials Ltd. to conduct clinical trials involving recombinant influenza viruses produced through gene technology. These studies aim to investigate viral infections and the development of immunity. These trials also seek to evaluate methods for preventing and controlling influenza, including the effectiveness of new drugs or vaccines^[⁴^].

INTERNATIONAL REGULATORY UPDATES RELEVANT TO AUSTRALIA & NEW ZEALAND

United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA)

On 30^th April 2025, the MHRA approved an alternative administration option for the cancer therapy drug nivolumab (OPDIVO), allowing for it to be given to eligible patients as a subcutaneous injection as opposed to an intravenous infusion. Nivolumab is a monoclonal antibody that binds to programmed death-1 (PD-1) on the patient’s T-cells and is used to treat a range of cancers. The approval was based on a randomized, open-label Phase 3 clinical trial in patients with advanced or metastatic clear cell renal cell carcinoma. The vast difference in timing of administration (3-5 minutes for the subcutaneous injection versus a 30- or 60-minute IV) will improve patient access and reduce pressure on healthcare services^[⁵^].

Following FDA approval of AUCATZYL (see ANZ LRA Watchdog December 2024), the MHRA have granted a conditional marketing authorization (CMA) of the CAR-T cell therapy to treat adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), following the FELIX study, an ongoing open-label, single-arm study that showed 52 out of 94 patients with the disease achieved complete remission and an 81% probability of overall survival at 12 months. A CMA is a temporary license, granted with fewer completed clinical studies than required for a full marketing authorization^[⁶^].

Also in the UK, on 23^rd July 2025, the new Human Medicines (Amendment) (Modular Manufacture and Point of Care) Regulations 2025, which will amend the Human Medicines Regulations 2012 and the Medicines for Human Use (Clinical Trials) Regulations 2004, will come into effect. Modular manufacture (MM), where products are manufactured in a modular unit allowing deployment from that location, and point of care (POC) manufacture, where products need to be manufactured close to where they are administered (due to characteristics like short shelf life, or being highly personalized to the patient), are together referred to as decentralized manufacture (DM). These regulations will provide clarity and guidance on regulatory expectations for DM around GCP, GMP, labelling, designation and both clinical trial and marketing authorization applications. It will enable DM by incorporating the adjustments needed to current regulations due to the dynamic aspects of manufacturing sites^[⁷^,⁸^].

United States Food & Drug Administration (FDA)

The US FDA are seeking comments on two guidance documents: Recommendations to Reduce the Risk of Transmission of Disease Agents Associated with Sepsis by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps)’ and ‘Recommendations to Reduce the Risk of Transmission of Mycobacterium tuberculosis (Mtb) by Human Cells, Tissues and Cellular and Tissue-Based Products (HCT/Ps). The first document provides recommendations for donor eligibility when screening a donor for clinical evidence of sepsis and what clinical signs should be considered. The second document addresses recommendations for screening donors for evidence of Mtb infection as well as risk factors. It also recommends additional steps HCT/P establishments should take to reduce Mtb transmission until FDA-approved donor screening tests are available. Comments for both documents are due by 7^th July 2025^[⁹^,¹⁰^]. These documents were previously released in January 2025 as final guidance but have been reissued as drafts based on feedback^[¹¹^].

In May 2025, the FDA announced they will expand the use of unannounced inspections for foreign manufacturers of foods, medicines and medical products destined for consumers and patients in the US, who generally receive notice of inspections unlike those in the US who undergo frequent unannounced inspections, to ensure those companies located overseas undergo the same level of regulatory oversight as those located domestically. The FDA’s policies and practices for the foreign inspection program will also be evaluated for best practice^[¹²^].

On 29^th April 2025 it was announced that the FDA approved ZEVASKYN (prademagene zamikeracel), the first autologous cell-based gene therapy for treating wounds in both adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is an incurable genetic disease, with those affected having extremely fragile skin with extensive blistering and severe wounds across the body that are difficult to heal, impacting quality and length of life. ZEVASKYN is the only FDA approved product for treatment, consisting of a sheet of the patients own skin cells that are genetically modified and produce function Type VII collagen. It is a singular surgical application and was shown in a Phase 3 multi-center, randomized clinical trial to provide both wound healing and pain reduction^[¹³^].

European Medicines Agency (EMA)

The EMA will make effective the ‘Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials – Scientific Guideline’ on 1^st July 2025. This guideline has undergone many rounds of consultation since 2018 and outlines the structure and data requirements for exploratory and confirmatory clinical trial applications for ATMPs. In the document, ATMPs are categorized as 1) gene therapy medicinal products for genetic disorders and cancers, 2) cell therapies including stem cells, and 3) tissue engineered products (e.g. skin grafts, artificial organs). It encompasses risk assessment, documentation, and process control in both manufacturing and quality. Importantly, it recommends that investigational ATMPs are produced according to Good Manufacturing Practices (GMP) and highlights the criticality of proper documentation and records when developing and manufacturing such products^[¹⁴^]. The guideline can be found here, and includes an overview of the comments that framed it’s development: https://www.ema.europa.eu/en/guideline-quality-non-clinical-clinical-requirements-investigational-advanced-therapy-medicinal-products-clinical-trials-scientific-guideline.

Japanese Society for Regenerative Medicine (JSRM)

The JSRM have published a paper in their Regenerative Therapy Journal, ‘Recommendations for the safe implementation of intravenous administration of mesenchymal stromal cells’ (MSCs), focusing on the safe IV administration of MSCs. Included is recommendations around potential risk and how they can be monitored for overall patient safety^[¹⁵^].

Foundation for the Accreditation of Cellular Therapy (FACT)

In April 2025, FACT announced their inaugural ‘Community CAR T Working Group’. Members of the group include community-based physicians who currently administer or plan to administer CAR T therapies, leaders from academic medical centers, and physician and clinical quality experts from FACT and other relevant professional societies. The aim of the group is to discuss challenges around patient access to such therapies and propose clinical accreditation standards for providers of these therapies, aligning with the upcoming third edition of the FACT Standards for Immune Effector Cells^[¹⁶^].

World first personalized CRISPR-based treatment in human

An infant suffering from the rare genetic condition carbamoyl phosphate synthetase 1 (CPS1) deficiency, where byproducts from protein metabolism in the liver cannot be fully broken down leading to ammonia buildup, has been successfully treated with a personalized gene editing therapy. Treatment options for this life threatening and incurable condition are usually a low protein diet until the patient is old enough for a liver transplant, but organ failure post-infection or trauma is common. The therapy was developed using the gene editing platform CRISPR by teams at the Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania (Penn) and is the first time this technology has been used to correct a specific genetic sequence in a human patient. The process from diagnosis to treatment took just six months and three doses, and corrected the specific gene mutation in the infant’s liver cells^[¹⁷^,¹⁸^].