ANZ LRA Watchdog – December 2024

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Kasey Kime
Director, Regulatory Affairs, Thermo Fisher Scientific

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

AUSTRALIAN AND NEW ZEALAND REGULATORY UPDATES

AOTEAROA – NEW ZEALAND

Medsafe

Regulatory collaboration and reliance can play a pivotal role in enhancing access to medicines, particularly in regions that may lack specific regulatory frameworks for Advanced Therapy Medicinal Products (ATMPs). While countries such as Australia, through the ACCESS Consortium, and the European Union, via the OPEN initiative, have established various collaboration mechanisms, there is still limited information available on similar pathways in New Zealand or Medsafe’s future plans for regulatory convergence.

Medsafe is currently developing a new reliance pathway, which has received Cabinet approval. This pathway will allow Medsafe to authorize new medicines that have already been approved by at least two other recognized regulatory authorities. The pathway will involve specific inclusion and exclusion criteria, such as limits on the time since initial approval and the exclusion of certain first-in-class products or those with unique national requirements, like vaccines. For further details, official documentation is available: https://www.health.govt.nz/system/files/2024-07/cabinet_and_briefing_material_streamlining_the_medicines_approvals_process_black_box_watermarked_to_publish.pdf.

AUSTRALIA

Therapeutic Goods Administration (TGA)

The TGA is seeking feedback on whether certain international guidelines should be adopted. A total of 19 are being considered, including ICH Q5A(R2) Guideline on viral safety evaluation of

biotechnology products derived from cell lines of human or animal origin, which describes the evaluation of the viral safety of biotechnology products such as biotherapeutics and biological products derived from cell lines from humans. Comments are open, via survey, until 16 January 2025: https://consultations.tga.gov.au/tga/international-scientific-guidelines-r01-2024/.

The South African Health Products Regulatory Authority (SAHPRA) and the TGA have signed a Memorandum of Understanding to strengthen the existing collaboration between the two regulators and build on the existing relationship to improve capabilities in the assessment and approval, and monitoring for efficacy/safety/quality, of medical products and therapeutic goods. The two will share data in an effort to improve the regulatory functions they oversee and expand their geographical reach [1].

The TGA released their annual stakeholder survey report on 14 November 2024. The survey is conducted to improve how the TGA works with stakeholders, report on key performance indicators and derive data to drive improvement. The survey had mostly positive results with regards to how the TGA performs its role ethically and with integrity, how the TGA balances access and safety, and responses to communications. It did however show a drop in satisfaction from opt-in stakeholders. The report can be accessed here: https://www.tga.gov.au/resources/publication/publications/tga-stakeholder-survey-report-2024.

The TGA Forum 2024 was held in Melbourne, Australia 19-20 November 2024, offering attendees the latest information, trends, issues and best practices in good manufacturing practice along with advances in PIC/S and Guide to GMP. Presentations will be made to available to registered delegates via email.

Office of the Gene Technology Regulator (OGTR)

The OGTR have released their Annual Report 2023-24 which describes their performance against deliverables and key performance indicators for the year. It lists licenses issued, breaches of licenses and auditing and monitoring. See here: https://www.ogtr.gov.au/sites/default/files/2024-10/operations_of_the_gene_technology_regulator_annual_report_2023_24.pdf.

INTERNATIONAL REGULATORY UPDATES RELEVANT TO AUSTRALIA & NEW ZEALAND

United Kingdom (MHRA)

A new Statutory Instrument laid in Parliament introduces a groundbreaking regulatory framework to facilitate the rapid and safe production of highly personalized and short-shelf-life medicines near hospitals, improving patient access. This regulation enables innovative manufacturing in small, mobile units close to patients, including those too ill or immunocompromised to travel, supporting treatments like cell and gene therapy. It is the first of its kind globally, enhancing the UK’s attractiveness for medical innovation. The framework also supports the expansion of ‘hospital at home’ initiatives, easing hospital burdens. The MHRA will provide comprehensive guidance and collaborate with stakeholders to ensure a smooth implementation by Summer 2025. See here: Statutory Instrument laid in Parliament provides first regulatory framework of its kind that will transform the manufacture of innovative medicines at the point of patient care - GOV.UK

United States (FDA)

The US FDA has released their Strategy Document draft for Innovative Technologies here: https://www.federalregister.gov/documents/2024/09/12/2024-20665/the-food-and-drug-administrations-draft-strategy-document-on-innovative-manufacturing-technologies.

They are seeking comments on their overall plan which includes several new initiatives for providers of Advanced Manufacturing Technologies and clearer guidance and timelines for achieving an Advanced Manufacturing Technology designation.

The FDA are also seeking feedback on a draft guidance document titled “Frequently asked Questions—Developing Potential Cellular and Gene Therapy Products”. This document is designed to provide answers to frequently asked questions around development of cellular and gene therapy products including regulatory review and manufacturing. Comments can be submitted until 18 February 2025 and the document can be found here: https://www.federalregister.gov/documents/2024/11/19/2024-26918/frequently-asked-questions-developing-potential-cellular-and-gene-therapy-products-draft-guidance.

On 8 November 2024, the FDA approved AUCATZYL (obecabtagene autoleucel, Autolus Inc.), a CD19-directed genetically modified autologous T cell immunotherapy, for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). It was granted regenerative advanced therapy designation and orphan drug designation, and the review used the ‘Assessment Aid’, a voluntary submission from the applicant to facilitate the assessment by the FDA [2]. AUCATZYL is the first CAR T therapy approved from the FDA with no requirement for a Risk Evaluation Mitigation Strategy (REMS) program, which may reduce burden for treatment centers in terms of ongoing reporting [3].

On 14 November 2024, the FDA approved KEBILIDI (eladocagene exuparvovec-tneq, PTC Therapeutics), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency – the first FDA approved gene therapy for this disease, and the first US gene therapy administered directly in the brain [4]. KEBILIDI was approved using the “Accelerated Approval Pathway”, where evidence of effectiveness was based on early improvements in gross motor function measured 48 weeks post treatment. The application received priority review and orphan drug designation, and was granted a rare pediatric disease priority review voucher [5].

Foundation for the Accreditation of Cellular Therapy (FACT)

FACT have released the 8^th edition of the NetCord-FACT International Standards for Cord Blood Collection, Banking and Release for Administration. FACT standards are based on evidence-based science and accepted scientific theory with emphasis on clinical outcomes. They apply to both family banking and cord blood for unrelated use. The updated standards are effective from 30 November 2024, and a cross walk and summary of changes is on the FACT website [6]. FACT have a free webinar, presented by A/Prof. Ngaire Elwood accompanied by Dr. Phyllis Warkentin, providing a comprehensive overview of the critical changes and implications for cord blood banks. The recording can be accessed here: https://learn.factglobal.org/2024-on-demand-webinar-cord-blood-standards-8th-edition.

FACT is also inviting public review and comment on the drafts of the 9^th edition of the FACT-JACIE International Standards for Hematopoietic Cellular Therapy Product Collection, Processing, and Administration (HCT Standards) and the 3^rd edition of the FACT-JACIE International Standards for Immune Effector Cells (IEC Standards). Comments will be accepted through to 15 December 2024 [7].

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