ANZ LRA Watchdog - August 2023

Recommend

Michelle Hudson¹ and Kasey Kime²
¹Clinical Trials Practice Educator, Parkville Cancer Clinical Trials Unit, Australia
²Director of Regulatory Affairs, Thermo Fisher Scientific, New Zealand

.

NEW CELL AND GENE THERAPIES APPROVED INTERNATIONALLY

It has been a busy two months with several new cell and gene therapies approved globally:

Product name	Generic name	Date of approval	Disease	Location & Regulator	Company
Gene therapies
Elevidys[AE1]	delandistrogene moxeparvovec	22 June 2023	Duchenne muscular dystrophy	US FDA	Sarepta Therapeutics
Roctavian	Valoctocogene roxaparvovec	29 June 2023	Severe Haemophilia A	US FDA	BioMarin Pharmaceutical Inc.
Cell therapies
Lantidra	donislecel	28 June 2023	Type 1 diabetes	US FDA	CellTrans
FUCASO	Equecabtagene Autoleucel	02 July 2023	Multiple myeloma	China NMPA	Innovent Biologics & IASO Bio

Although these therapies are currently unavailable in Australia or New Zealand, their international approvals by comparable overseas regulators could expedite the evaluation process by local regulators. As a result, access to these therapies in the future may be faster.

.

WATCH THIS SPACE

BioCardia announcement on July 05, 2023: “BioCardia Completes Submission of CardiAMP Cell Therapy System in Heart Failure to Japan’s Pharmaceutical Medical Device Agency

BioCardia®, Inc. [Nasdaq: BCDA], a developer of cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary diseases, today announces it completed its submission of the CardiAMP Cell Therapy System to Japan’s Pharmaceutical and Medical Device Agency (PMDA) towards approval for the indication of ischemic heart failure with reduced ejection fraction (HFrEF[DM1] [PA2]). The formal consultation with PMDA to review the submission is anticipated in approximately three months and will be BioCardia’s third consultation with PMDA” (1).

Singapore Company, Biosyngen, Receives FDA Fast Track Designation

“BRG01, an immunotherapy drug developed by Biosyngen, has received regulatory approvals from both the China National Medical Products Administration (NMPA) on December 14, 2022, and the U.S. Food and Drug Administration (FDA) on February 16, 2023, respectively; as an Investigational New Drug (IND) for the treatment of relapsed/metastatic nasopharyngeal carcinoma. Further to that, it was granted Orphan Drug Designation (ODD) by the FDA on June 1, 2023. With this latest news, FDA's granting of Fast Track Designation to BRG01 signifies a significant acknowledgment of Biosyngen's advanced technology and product, demonstrating Biosyngen's strong commitment to developing groundbreaking products with a global market focus” (2).

Feedback sought on proposed changes to genetically modified organisms regulations in New Zealand

The Government is seeking feedback on proposed changes to Aotearoa New Zealand’s legislation and regulations for genetically modified organisms (GMOs). This is when they are used in laboratory settings and for biomedical therapies.

The proposed changes respond to concerns from the research community that current GMO regulations are hindering research and innovation. They aim to remove barriers to foster research and improve health outcomes (3).

.

REGULATORY INTELLIGENCE UPDATES

Therapeutic Product Bill NZ Update

In June the Bill passed its second reading. There were over 16,000 submissions regarding the Bill. However, the Health Committee was unable to reach an agreement on whether to recommend the Bill be passed. Interestingly, an amendment will be made to the definition of a biologic (Clause 32) to include microorganisms within the definition of a biologic. This would mean that viral vector-based gene therapies would be considered biologics in New Zealand which is aligned to international classifications (USA and EU) but is not aligned to Australia whereby viral vector gene therapies are currently regulated as prescription medicines not biologics (4).

ICMRA statement of the safety of COVID-19 vaccines

This International Coalition of Medicines Regulatory Authorities (ICMRA) statement provides the general public with important information regarding the safety of COVID-19 vaccines, which have now been in use for more than two years. It also addresses some of the most common types of misinformation about COVID-19 vaccine safety (5).

New US FDA Guidance for Manufacturing Changes and Comparability for Human Cellular and Gene Therapy Products

The USA FDA has released draft guidance for comment regarding the management of manufacturing changes that occur in the IND and commercial phases. This is the first CGT-specific guidance for manufacturing changes made during clinical trial phases and is a welcome addition to extensive FDA guidance’s for CGT drug development (6). This guidance is relevant to Australian clinical trials that may be performed under an open US FDA IND.

The new OGTR Online Services Portal is now available

The Office of the Gene Technology Regulator has launched a new online portal for managing and tracking applications. The OGTR Online Services Portal can be used to draft, report, and view Notifiable Low Risk Dealings (NLRDs). In the near future, the portal will enable services such as applications for certification and contact management, and eventually other application types. The OGTR will provide communication regarding access to these services in due course (7).

AusBiotech launches National Cell and Gene Manufacturing Blueprint

Supported by the Victorian Government’s Australian Medtech Manufacturing Centre (AMMC), the Blueprint was developed by AusBiotech, the Cell and Gene Manufacturing Taskforce, and Biointelect with both industry and government in mind. It addresses five key areas outlined in the Strategic Roadmap for the Regenerative Medicine Sector: workforce skills development, long-term investment opportunities, strengthened collaboration across the value chain, capability across the value chain, and clear market access pathways aligned with leading global markets (8). The blueprint as well as the abovementioned strategic roadmap both had significant input from a number of ISCT ANZ members across a range of disciplines, highlighting how ISCT members are shaping the future of Cell and Gene Manufacturing in Australia and New Zealand. A full list of contributors can be found on page 61-62 of the National Cell and Gene Manufacturing Blueprint and pages 2-4 of the Strategic Roadmap.

.

OTHER NEWS:

The critical shortage of heparinised saline 50 units/5mL and Heparin Sodium 5000IU/5ml

Heparinised saline 50IU/5mL ampoules are in critical shortage across Australia (9). Some stock is due to return at the end of July 2023, but supplies are expected to remain limited until at least mid-July 2023 and the shortage may continue beyond. The anticoagulant medication has a range of applications ranging from central line management and thrombosis prevention to some specialised apheresis and cryopreservation applications. An unregistered product has been approved for supply under section 19A of the Therapeutic Goods Act. Consult local risk assessment and mitigation guidelines within your institution (10).

.

REFERENCES