Australia and New Zealand LRA Watchdog Report

Australia and New Zealand LRA Watchdog Report – October 2025

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Kasey Kime, MBA
BioOra
New Zealand

Giulia Giunti, PhD
BioOra
New Zealand

AOTEAROA – NEW ZEALAND

Medsafe

Medsafe published their performance statistics for the evaluation of new and changed medicines between 1 July 2024 and 30 June 2025 (1).

Ministry of Business, Innovation & Employment (MBIE) and Environmental Protection Authority (EPA)

The Gene Technology Bill is currently being considered by Parliament’s health select committee, which is due to report back 10 October 2025 (2).

AUSTRALIA

TGA

The TGA adopted following 3 international scientific guidelines in September 2025 (3):

Office of the Gene Technology Regulator (OGTR)

The OGTR has granted a license (DIR 213, issued June 2025). This licence allows Novotech Australia Pty Ltd. to conduct clinical trials involving a genetically modified human adenovirus for the treatment of melanoma (4).

INTERNATIONAL REGULATORY UPDATES RELEVANT TO AUSTRALIA AND NEW ZEALAND

European Medicines Agency (EMA)

EMA has issued a grant to the European Directorate for the Quality of Medicines & HealthCare (EDQM), Council of Europe, who will contribute its expertise in medicines quality control to a major EU initiative supporting regulatory systems across Sub-Saharan Africa. This work supports the operationalisation of the African Medicines Agency and the wider goal of building resilient regulatory systems that ensure access to safe, effective and high-quality medicines across Africa (5).

United States (FDA)

The FDA has published a series of guidance documents aimed at improving the regulatory framework for cell and gene therapies, including three draft guidance’s released in late 2025 focused on post-approval methods for safety and efficacy data, considerations for clinical trials in small populations, and the expedited programs for regenerative medicine (6).

On September 24, 2025, the FDA released a new guidance on “Computer Software Assurance for Production and Quality System Software”, which introduces the use of risk-based assurance (7).

RMAT Tracker for September 2025

Nanoscope Therapeutics has received FDA regenerative medicine advanced therapy (RMAT) designation for MCO-010 (sonpiretigene isteparvovec), an investigational ambient-light activatable multicharacteristic opsin (MCO) gene therapy intended to treat various retinal diseases. The RMAT designation specifically applies to Stargardt disease. Notably, the European Medicines Agency has also granted MCO-010 orphan drug designation for 5 retinal dystrophy categories, including macular dystrophies and nonsyndromic and syndromic rod-dominant and cone-dominant dystrophies. The FDA has also previously granted MCO-010 orphan drug designation and fast track designation (8).
Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe neurological disorders, announced the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ETX101 following review of preliminary seizure data from patients treated in its ongoing Phase 1/2 program. ETX101 is an AAV9-based gene regulation therapy designed to increase SCN1A expression in GABAergic inhibitory neurons for the treatment of SCN1A+ Dravet syndrome. ETX101 has Fast Track, Rare Pediatric, and Orphan Drug Designations from FDA and Orphan Designation from EMA (9). Europe (EMA)

Reminder that Stakeholders’ Consultation on EudraLex Volume 4 - Good Manufacturing Practice Guidelines: Chapter 4 (Documentation), Annex 11 (Computerised Systems) and New Annex 22 (Artificial Intelligence) will close on 7^th October 2025 (10).

Iovance Therapeutics has withdrawn their market authorization application for Amtagvi (lifileucel), a medicine to treat melanoma that is unresectable (cannot be removed by surgery) or metastatic (has spread to other parts of the body) in adults. Based on the review of the data and the company’s response to the Agency’s questions, at the time of the withdrawal, the Agency had some concerns and its provisional opinion was that Amtagvi could not have been authorised for the treatment of melanoma.

The Agency had concerns about the response to Amtagvi in the main study, which was too low to determine whether treatment with Amtagvi would provide a meaningful benefit to patients. There were also concerns about the safety of the Amtagvi treatment regimen due to serious side effects, which resulted in death in some cases.

The Agency also noted that the data to support the proposed dose were not sufficient. In addition, the applicant had not provided all the necessary good manufacturing practice (GMP) documentation (11).

United Kingdom MHRA

The MHRA has updated the decentralized manufacturing homepage with a new article titled: Decentralised Manufacturing: emerging considerations. This article highlights the UK’s new Decentralised Manufacturing (DM) framework, enabling point-of-care and modular manufacturing to improve patient access to personalised medicines while ensuring quality and safety. DM requires MHRA designation with evidence of clinical benefit, GMP oversight, and inspections, and aligns with similar initiatives in the EU and US (12).

International Society for Pharmaceutical Engineers (ISPE)

This ISPE has published ISPE Guide: ATMPs – Validation Methods & Controls which offers practical guidance on applying validation methods and control throughout the cell and gene therapy product lifecycle. It outlines risk-based strategies tailored to the unique nature of these products, addressing key areas such as analytical methods, equipment, processes, cleaning, and supply chain. It also includes essential insights into regulatory expectations and phase-appropriate considerations that support timely development and commercialization (13).

Parenteral Drug Association (PDA)

The PDA has issued Technical Report 13, New Points to Consider on Materials in ATMP Manufacturing. This Q&A style report provides critical guidance for managing raw materials in the production of advanced therapy medicinal products (ATMPs), aiming to support stakeholders in developing robust material control strategies and promoting best practices across the industry (14).

BioPhorum Operations Group (BPOG)

BioPhorum has compiled a set of frequently asked questions addressing the scope, applicability and global interpretation of Annex 1 (15). This guide clarifies:

What Annex 1 is and where it applies
Its relevance to sterile and non-sterile operations
Differences between EU, PIC/S and WHO versions
How regulators like the FDA are referencing Annex 1 in inspections
The rationale behind the 2022 revision and its alignment with ICH Q9/Q10.

International Pharmaceutica Regulators Programme (IPRP)

The Cell and Gene Therapy Working Group published the International Regulatory Frameworks for Cell and Gene Therapies, dated July 2025 (16).

International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH)

In follow up to the publication of the ICH Q3E draft “Guideline for Extractables and Leachables” in August 2025, the Q3E EWG has also prepared a Step 2 informational presentation.

The Q3E Step 2 presentation is now available for download on the Q3E EWG Page (17).