Mina Hosseini, PhD, Pharm.D, MPH
Member, ISCT ANZ LRA Committee
Junior Associate Editor, ISCT Telegraft
Australia
AUSTRALIAN AND NEW ZEALAND REGULATORY UPDATES
TGA
TGA guidance: manufacturing, supplying and advertising compounded medicines lawfully
The TGA has published new guidance outlining the regulatory requirements for the lawful manufacture, supply and advertising of compounded medicines in Australia. The guidance clarifies when exemptions from Australian Register of Therapeutic Goods (ARTG) inclusion and manufacturing licensing requirements may apply, while emphasising that compounding should only occur where necessary to meet the clinical needs of an individual patient. It also outlines additional regulatory expectations for higher-risk products, including biologicals, medicinal cannabis, GLP-1 receptor agonists and intravenous therapies. The publication aims to promote regulatory clarity, support compliance across the health sector, and ensure the safe and lawful use of compounded medicines.
LINK: https://www.tga.gov.au/resources/guidance/manufacturing-supplying-and-advertising-compounded-medicines-lawfully
TGA updates authorised prescriber requirements for MDMA and psilocybin therapies
The TGA has published the outcomes of a targeted consultation reviewing Authorised Prescriber (AP) scheme requirements for MDMA and psilocybin-assisted psychotherapy. The recommendations clarify psychiatrist competency pathways, therapy team composition, prescriber oversight responsibilities, and minimum treatment site requirements. The updated framework aims to support safe and consistent access to psychedelic-assisted therapies while maintaining appropriate clinical governance and patient safety safeguards.
LINK: https://consultations.tga.gov.au/medicines-regulation-division/targeted-consultation-ap-scheme-for-psychedelics/
TGA updates guidance on Unique Device Identification (UDI) compliance timeframes
The TGA has updated its guidance on compliance timeframes for Australia's Unique Device Identification (UDI) framework for medical devices and IVDs. The revised guidance clarifies transitional arrangements, sponsor responsibilities, treatment of existing devices, consignment stock, surgical loan kits, and the transition from EU MDD/IVDD to MDR/IVDR classifications. The update aims to assist manufacturers and sponsors in preparing for the phased implementation of UDI requirements and meeting future compliance deadlines.
LINK: https://www.tga.gov.au/resources/guidance/complying-unique-device-identification-timeframes-medical-devices#page-history
MEDSAFE
Medsafe warns against importation and use of unapproved peptide products
Medsafe has issued a safety advisory highlighting increasing imports and sales of unapproved peptide products and selective androgen receptor modulators (SARMs) in New Zealand. The regulator warned that many products are poorly labelled, may contain undisclosed ingredients or contaminants, and have not been assessed for quality, safety, or efficacy. Medsafe noted that such products are frequently marketed online under misleading claims, including “for research purposes only”, and reiterated that the importation, supply, and use of many of these products is unlawful outside approved regulatory pathways. The advisory reflects increasing regulatory scrutiny across Australia and New Zealand regarding the importation and online promotion of unapproved peptide products.
LINK: https://www.medsafe.govt.nz/safety/Alerts/Consumer-advisory-Unapproved-peptide-products-health-warning.asp
Medsafe MARC recommends review of vitamin B6 regulation
At its March 2026 meeting, New Zealand's Medicines Adverse Reactions Committee (MARC) reviewed safety concerns relating to pyridoxine (vitamin B6), particularly the risk of peripheral neuropathy associated with chronic exposure. The committee noted Australia's recent decision to tighten controls on higher-dose vitamin B6 products and recommended that New Zealand regulators consider reducing the amount of pyridoxine permitted in general-sale medicines and dietary supplements, alongside introducing warning statements. The recommendations have been referred to both the Medicines Classification Committee and the Ministry for Primary Industries for further consideration.
LINK: https://www.medsafe.govt.nz/profs/adverse/Minutes205.htm
AUSTRALIAN GOVERNMENT
Australia strengthens protections against genetic discrimination in life insurance
The Treasury Laws Amendment (Genetic Testing Protections in Life Insurance and Other Measures) Act 2026 came into force on 8 April 2026, preventing life insurers from using certain genetic test information to determine eligibility for cover, premiums, or policy conditions. The reform is intended to support access to genetic testing and encourage participation in medical research by reducing concerns about potential insurance discrimination. The legislation represents a significant development in Australia's approach to genetic privacy and may be of particular interest as genomic medicine, precision medicine, and gene-based therapies continue to expand across the healthcare sector.
LINK: https://parlinfo.aph.gov.au/parlInfo/download/legislation/ems/r7409_ems_eb970c25-aef4-4954-8a94-adc8d4050633/upload_pdf/JC017193.pdf;fileType=application%2Fpdf
INTERNATIONAL REGULATORY UPDATES RELEVANT TO AUSTRALIA & NEW ZEALAND
FDA
FDA approves first gene therapy for OTOF-associated genetic hearing loss
The FDA approved Otarmeni (lunsotogene parvec-cwha) on 23 April 2026 for the treatment of severe-to-profound sensorineural hearing loss associated with biallelic OTOF mutations. The product is the first approved dual-AAV vector gene therapy and the first gene therapy authorised through the FDA's Commissioner’s National Priority Voucher (CNPV) pilot programme. Approval was granted under the accelerated approval pathway based on data demonstrating clinically meaningful hearing improvement in patients with OTOF-related deafness, a condition for which no disease-modifying therapies were previously available.
The subsequently published Summary Basis for Regulatory Action (SBRA) provides additional insight into the FDA’s benefit-risk assessment, clinical evidence package, manufacturing considerations and accelerated review process. The approval highlights continued regulatory support for innovative gene therapies targeting rare genetic disorders and demonstrates the FDA’s willingness to use expedited review mechanisms for transformative cell and gene therapy products addressing significant unmet medical needs.
LINK: https://www.fda.gov/news-events/press-announcements/fda-approves-first-ever-gene-therapy-treatment-genetic-hearing-loss-under-national-priority-voucher
FDA Finalizes CMC Flexibility Guidance for Human Cellular and Gene Therapy Products
The FDA published a new guidance, "Chemistry, Manufacturing, and Controls (CMC) Flexibilities for Developing Human Cellular and Gene Therapy Products for a Biologics License Application" (May 2026), outlining how the agency applies regulatory flexibility to the requirements for CGT products. The guidance acknowledges the unique manufacturing challenges associated with CGTs and clarifies circumstances where flexibility may be appropriate while maintaining product quality, safety, and efficacy standards. FDA states that this risk-based approach is intended to facilitate development, regulatory review, and patient access to CGT products targeting serious or life-threatening diseases with significant unmet medical need.
LINK: https://www.fda.gov/media/192321/download
FDA publishes updated ICH Q8, Q9 and Q10 Questions & Answers (R5) guidance
The FDA has issued the ICH Q8, Q9 and Q10 Questions and Answers (R5) guidance, providing additional clarification on the implementation of the pharmaceutical quality system framework established by "ICH Q8 (Pharmaceutical Development)", "Q9 (Quality Risk Management)" and "Q10 (Pharmaceutical Quality System)". The update reflects practical experience gained across ICH regions since the original guidance was adopted and addresses areas where industry and regulators have sought greater clarity. The guidance is intended to promote more consistent application of quality-by-design principles, risk-based decision making, and lifecycle quality management across pharmaceutical and biologic product development and manufacturing.
LINK: https://www.fda.gov/media/78668/download
FDA seeks stakeholder input on drug repurposing for unmet medical needs
The FDA has opened a public consultation to identify opportunities for drug repurposing, particularly in areas of unmet medical need such as rare diseases, neurodegenerative disorders, and metabolic conditions. The agency is seeking input on approved drugs that may support new indications based on existing clinical, preclinical, or real-world evidence, with the goal of accelerating patient access to therapies where commercial incentives for further development may be limited.
LINK: https://www.fda.gov/news-events/press-announcements/fda-advances-drug-repurposing-address-unmet-medical-needs
FDA announces OTP Town Hall on BLA readiness for cell and gene therapies
The FDA's Office of Therapeutic Products (OTP) will host a virtual town hall on 4 June 2026 focused on best practices for preparing "Biologics License Applications (BLAs)" for CGT products. The session will address BLA readiness, pre-BLA meeting requests, submission package preparation, and navigation of the marketing application process. The event forms part of FDA's ongoing engagement with product developers and provides insight into current regulatory expectations for CGT licensure.
LINK: https://www.fda.gov/vaccines-blood-biologics/news-events-biologics/otp-town-hall-best-practices-preparing-bla-submissions-cell-and-gene-therapy-products-06042026
FDA, MHRA and Health Canada to host joint symposium on clinical trials and pharmacovigilance
FDA, MHRA and Health Canada will jointly host a regulatory symposium on 2–4 June 2026 covering good clinical practice (GCP), bioequivalence and pharmacovigilance. Planned topics include implementation of ICH E6(R3), quality-by-design approaches to clinical trials, decentralized and pragmatic trial designs, integration of real-world data, bioequivalence assessment, and international pharmacovigilance inspection practices.
LINK: https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/fdamhrahealth-canada-symposium-regulatory-perspectives-good-clinical-practice-bioequivalence-and
FDA workshop highlights development of novel surrogate endpoints for rare diseases
On 18 May 2026, the FDA convened a public workshop on the development of novel surrogate endpoints for rare disease therapies as part of commitments under PDUFA VII and the Food and Drug Omnibus Reform Act. Discussions focused on the evidentiary requirements needed to support surrogate endpoints in rare disease marketing applications, including mechanistic and translational evidence, digital health technologies, novel methodologies and the use of real-world patient data.
LINK: https://www.fda.gov/drugs/news-events-human-drugs/advancing-novel-surrogate-endpoints-rare-disease-drug-development-workshop-05182026
FDA hosts REdI 2026 conference on innovative regulatory strategies
The FDA's annual Regulatory Education for Industry (REdI) conference was held on 19–20 May 2026, bringing together experts from CDER, CBER and CDRH. Topics included artificial intelligence in drug development, advanced manufacturing, digital health technologies in clinical trials, benefit-risk assessment, and regulatory considerations for biologics, devices and medicines. Recordings of the sessions have been made publicly available.
LINK: https://www.fda.gov/news-events/regulatory-education-industry-redi-annual-conference-2026-innovative-regulatory-strategies-advance
EMA
EMA grants PRIME eligibility to four innovative medicines, including three advanced therapies
The European Medicines Agency (EMA) published the outcomes of its April 2026 review of applications to the "PRIority MEdicines (PRIME)" scheme. Of ten applications assessed by the Committee for Medicinal Products for Human Use (CHMP), four were granted PRIME eligibility and six were denied. The successful applications included three advanced therapy medicinal products (ATMPs): zertomibgene cazparvovec for hypertrophic cardiomyopathy caused by MYBPC3 mutations, an adeno-associated virus (AAV9)-based gene therapy (TN-401) for arrhythmogenic right ventricular cardiomyopathy associated with PKP2 mutations, and an AAV2.7m8-based gene therapy for retinitis pigmentosa. A fourth product, efimosfermin alfa, received PRIME eligibility for the treatment of metabolic dysfunction-associated steatohepatitis (MASH).
The predominance of gene therapy products among successful applicants underscores EMA’s ongoing support for innovative therapies addressing serious conditions with unmet medical needs. PRIME designation provides developers with enhanced regulatory support and early scientific interaction with EMA to facilitate the development and accelerated assessment of promising medicines.
LINK: https://www.ema.europa.eu/en/documents/chmp-annex/recommendations-eligibility-prime-scheme-adopted-chmp-meeting-20-23-april-2026_en.pdf
EU reaches political agreement on Critical Medicines Act
The European Parliament and the Council of the European Union reached a provisional agreement on the Critical Medicines Act (CMA), a legislative initiative designed to strengthen the availability, production and supply security of critical medicines across the EU. The Act aims to address vulnerabilities in pharmaceutical supply chains exposed by recent medicine shortages, the COVID-19 pandemic and geopolitical disruptions.
The CMA complements the revised EU pharmaceutical legislation and expands the role of EMA in monitoring supply-chain vulnerabilities, supporting shortage prevention and strengthening manufacturing resilience. Key measures include enhanced use of the European Shortages Monitoring Platform (ESMP), supply-chain risk assessments for critical medicines, and support for innovative manufacturing technologies.
LINK: https://www.ema.europa.eu/en/news/ema-welcomes-political-agreement-critical-medicines-act
EU reports early progress toward 2030 clinical trial targets
The European Commission, EMA and the Heads of Medicines Agencies (HMA) published the first report tracking progress against the EU’s 2030 clinical trial targets. Covering the first quarter of 2026, the report found that 19 additional multinational clinical trials were authorised above the historical average, representing early progress toward the goal of 500 additional multinational trials by 2030.
The report also showed that 40.5% of clinical trials recruited participants within 200 days of application submission, compared with a target of 66% by 2030. The findings suggest continued improvement in the efficiency and attractiveness of the EU clinical research environment, supporting broader efforts under the ACT EU initiative and the proposed EU Biotech Act to accelerate the development of innovative medicines.
LINK: https://www.ema.europa.eu/en/news/eu-tracks-progress-towards-2030-clinical-trial-targets
ACT EU publishes revised 2026–2027 workplan
The "Accelerating Clinical Trials in the EU (ACT EU)" initiative published its revised 2026–2027 workplan, outlining priorities to strengthen the European clinical research environment. Key focus areas include optimisation of the "Clinical Trials Regulation (CTR)", maximising the impact of clinical trials, preparedness for public health emergencies, and enhanced use of clinical trial analytics.
New priorities include strengthening patient involvement throughout the clinical trial lifecycle, improving the use of clinical trial data to support access and inclusivity, and promoting timely submission of trial results. The updated workplan reflects stakeholder feedback and supports ACT EU’s goal of making the EU a more attractive and efficient environment for innovative clinical research.
LINK: https://www.ema.europa.eu/en/human-regulatory-overview/research-development/clinical-trials-human-medicines/accelerating-clinical-trials-eu-act-eu
EMA recommends XFG strain update for COVID-19 vaccines
For the 2026–2027 vaccination campaign, EMA’s Emergency Task Force (ETF) has recommended updating authorised COVID-19 vaccines to target the XFG SARS-CoV-2 variant. The recommendation is based on emerging epidemiological and immunogenicity data indicating that XFG-adapted vaccines potentially provide broad protection against currently circulating JN.1-related variants and the emerging BA.3.2 lineage. Marketing authorisation holders are expected to engage with EMA regarding strain-update variations to support vaccine deployment for the upcoming season.
LINK: https://www.ema.europa.eu/en/news/etf-recommends-updating-covid-19-vaccines-target-xfg-variant
EMA advances virtual control groups to reduce animal testing in drug development
EMA’s CHMP issued a draft qualification opinion supporting the use of virtual control groups in certain preclinical dose-range finding studies. The approach would replace concurrent animal control groups with statistically derived virtual comparators, potentially reducing the number of animals required in toxicology studies while maintaining scientific validity. The initiative represents EMA’s first qualification opinion for a New Approach Methodology (NAM) in toxicity assessment and may serve as a blueprint for broader regulatory adoption of alternatives to animal testing. Public consultation on the draft opinion closed on 12 May 2026.
LINK: https://www.ema.europa.eu/en/news/ema-consults-virtual-control-groups-help-reduce-animal-use-medicines-development
EMA Accelerated Assessment of Regeneron’s Gene Therapy Otarmeni for OTOF-Related Hearing Loss
EMA has accepted Regeneron’s Marketing Authorisation Application (MAA) for Otarmeni (lunsotogene parvec-cwha), for biallelic OTOF variant-associated severe-to-profound hearing loss, under Accelerated Assessment. Accelerated Assessment indicates the CHMP’s view that the therapy may be of major public health interest and enables a shortened review timeline compared to standard assessment procedures. The application is supported by data from a Phase 1/2 CHORD study conducted across multiple regions, including the US, Europe, and Asia, in a paediatric and young adult population. If approved, this would represent the first gene therapy authorised in the EU for OTOF-related hearing loss and aligns with the recent FDA approval of the same product, reflecting ongoing convergence in expedited gene therapy review pathways across major regulatory jurisdictions.
LINK: https://au.investing.com/news/stock-market-news/ema-accepts-regenerons-gene-therapy-for-rare-hearing-loss-93CH-4452074
ICH
ICH M11 Electronic Structured Protocol Standard (CeSHarP) Reaches Step 4 with Final Overview Presentation
The ICH Expert Working Group has issued the Step 4 final overview presentation for "ICH M11: The Clinical electronic Structured Harmonised Protocol (CeSHarP)", a guideline adopted in November 2025 aimed at standardising the format, content, and electronic exchange of clinical trial protocols. ICH M11 comprises a harmonised protocol guideline, a structured protocol template, and a technical specification enabling interoperable electronic transfer of protocol information across regulatory and sponsor systems. The guideline applies broadly to interventional clinical trials across all therapeutic areas, including pharmaceuticals, biologics, vaccines, drug-device combinations, and cell and gene therapies. Its implementation addresses longstanding variability in protocol design and exchange across regions and sponsors, with the objective of improving regulatory review efficiency, data searchability, and interoperability of clinical trial documentation within the global regulatory ecosystem.
LINK: https://ich.org/news/ich-m11-expert-working-group-issues-final-overview-presentation
JAPAN
Japan initiates reimbursement and clinical rollout of first iPSC-derived Parkinson’s therapy
Japan’s Ministry of Health, Labour and Welfare (MHLW) advisory panel has approved public insurance coverage for Sumitomo Pharma’s Amchepry, an induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cell therapy for Parkinson’s disease, with coverage effective from May 2026 and clinical availability expected later in the year. The decision marks a key reimbursement and health system integration milestone for an iPSC-derived therapy following prior regulatory clearance processes in Japan’s regenerative medicine framework. Amchepry is administered via intracerebral transplantation of donor-derived neural progenitor cells into the putamen, where they are intended to mature into dopamine-producing neurons to partially restore striatal dopaminergic signalling. The inclusion of the therapy in the national insurance program represents an important precedent for the reimbursement of high-cost regenerative medicine products and reflects Japan’s continued role as a leading jurisdiction for early clinical adoption and health technology integration of advanced cell-based therapies.
LINK: https://regmedfoundation.org/2026/05/27/japan-greenlights-public-coverage-for-ips-parkinsons-treatment/
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