Watchdog Update
June 2022
By:
Gabrielle O’Sullivan PhD MPH
Executive Officer, Royal Prince Alfred Hospital Institutional
Biosafety Committee, Royal Prince Alfred Hospital
Australia
Giulia Giunti PhD
Quality Manager, Malaghan Institute of Medical Research
Wellington, New Zealand
HAEMOPHILIA B INVESTIGATIONAL GENE THERAPY - CSL BEHRING BLA ACCEPTED BY FDA FOR PRIORITY REVIEW
On 24 May 2022, CSL Behring announced that the FDA has accepted its Biologics License Application (BLA) for priority review for etranacogene dezaparvovec (also known as CSL222), an investigational AAV-vector gene therapy for hemophilia B. CSL Behring is a global biotherapeutics business of Melbourne-based CSL Limited. It acquired the investigational gene therapy from the biotech uniQure in June 2020. The BLA is supported by results from the pivotal HOPE-B trial, in which those treated with etranacogene dezaparvovec demonstrated reduced adjusted annualized bleeding rate by 64% and superiority to prophylaxis treatment at 18 months post-treatment compared to a 6-month run in period (p=0.0002). [1, 2]
MEDSAFE APPROVALS
MedSafe has reported its approvals of new active ingredients and new indications for the period 16 January to 15 April 2022. Among them is the approval of Evrysdi (risdiplam), an RNA therapy for spinal muscular atrophy; and Nuvaxovid for COVID-19 prevention and Paxlovid and Lagevrio for COVID-19 treatment. [3]
AMENDMENTS TO MEDSAFE GUIDELINES
On 24 April 2022, MedSafe published minor amendments on 2 February 2022 to Part 4 of the “Guideline on the regulation of therapeutic products in New Zealand: Manufacture of Medicines”. The changes are: added requirement for GMP for API that are prescription medicines; added requirement for ongoing evidence of current compliance to be provided; updated logos for Romania, and Switzerland; updated websites for Canada, Hungary, Italy, Luxembourg, Romania, Singapore and Spain; removed reference to Part 5 of the New Zealand Regulatory Guidelines for Medicines (NZRGM). [4]
TGA
TGA presentations at the ARCS 2022 annual conference
Presentations given by the TGA at the 2022 Association of Regulatory and Clinical Scientists (ARCS) Annual Conference in Sydney, 23-25 May 2022 are now available online. These include a presentation on the regulation of cell and gene therapies in Australia. [5]
TGA Consultation on the adoption of EMA guideline on the use of patient-reported outcome (PRO) measures in oncology studies for the evaluation of anticancer medicinal products
The TGA regularly consults on the adoption of certain international scientific guidelines in Australia. While international scientific guidelines that are adopted in Australia are generally not mandated by legislation, they provide guidance to sponsors in meeting legislative requirements. Deviations from an adopted guideline relevant to an application to register or vary the registration of a medicine will need to be justified. Currently the TGA is consulting on whether or not to adopt the EMA guideline ‘Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man. The use of patient-reported outcome (PRO) measures in oncology studies (EMA/CHMP/292464/2014)’. The closing date for comments is 29 July 2022. [6,7]
The TGA’s transition period from the 2018 to the 2021 Therapeutic Goods Advertising Code ends on 30 June 2022
The transition period from the 2018 to the 2021 Therapeutic Goods Advertising Code ends on 30 June 2022. The 2021 Code came into effect on 1 January 2022 with a 6 month transition period to 30 June 2022. On 1 July 2022 all advertising, including on social media, must comply with the 2021 code. [8]
TGA guidance on the requirements regarding testimonials and endorsements in advertising
Section 24 of the Therapeutic Goods Advertising Code 2021 sets out the requirements for using endorsements and testimonials in advertisements about therapeutic goods. The TGA has produced guidance that explains the requirements and provides examples. [9]
TGA Evidence Guidelines
At the time of listing a medicine in the Australian Register of Therapeutic Goods (ARTG), sponsors must certify that they hold evidence to support any indications and claims made about their medicine. The TGA has published 'Evidence guidelines: How to demonstrate the efficacy of listed medicines is acceptable'. These specify the type of evidence required to support indications made for listed medicines (excluding sunscreens and listed assessed medicines) and help sponsors understand their regulatory obligations in relation to holding that evidence. [10]
OFFICE OF THE GENE TECHNOLOGY REGULATOR (OGTR)
The OGTR held its 9th National Institutional Biosafety Committee (IBC) Forum at the Shine Dome, Canberra on Thursday 12 May – Friday 13 May 2022. The Forum was well attended and celebrated the 20th year anniversary of the Australian Gene Technology Regulatory Scheme which came into effect on 21 June 2001. Among the topics discussed were clinical trials, community attitudes to gene technology and the progress of reforms to the Scheme. [11]
REFERENCES
[1] CSL Behring, Newsroom, FDA Accepts CSL Behring’s Biologics License Application for Etranacogene Dezaparvovec for Priority Review, https://www.cslbehring.com/newsroom/2022/fda-bla-etranacogene-dezaparvovec, published 24 May 2022, accessed 21 June 2022
[2] G Masson, Fierce Biotech, CSL Behring gene therapy makes comeback after hold for FDA priority review, https://www.fiercebiotech.com/biotech/2020-trial-pause-isnt-holding-csl-behring-back-fda-oks-priority-review-application, published 25 May 2022, accessed 21 June 2022
[3] MedSafe Prescriber Update, 43 (2), June 2022, https://www.medsafe.govt.nz/profs/PUArticles/PDF/Prescriber-Update-Vol-43-No.2-June-2022.pdf, accessed 21 June 2022
[4] MedSafe, https://www.medsafe.govt.nz/, https://www.medsafe.govt.nz/regulatory/current-guidelines.asp and https://www.medsafe.govt.nz/regulatory/Guideline/GRTPNZ/manufacture-of-medicines.pdf, 2 February 2022, published 24 April 2022, accessed 20 June 2022
[5] TGA presentations: 2022 ARCS Annual Conference, 23-25 May 2022, https://www.tga.gov.au/tga-presentations-2022-arcs-annual-conference-23-25-may-2022, published 21 June 2021, accessed 21 June 2021
[6] TGA, Adoption of international scientific guidelines in Australia, https://consultations.tga.gov.au/tga/rwe-proposed-adoption-of-international-scienti/, opened 20 Jun 2022, closes 29 July 2022
[7] TGA Consultation hub, https://consultations.tga.gov.au/tga/rwe-proposed-adoption-of-international-scienti/consultation/subpage.2021-06-22.1492661585/, closes 29 July 2022
[8] TGA, The transition period from the 2018 to the 2021 Advertising Code ends 30 June 2022, https://www.tga.gov.au/media-release/transition-period-2018-2021-advertising-code-ends-30-june-2022, published 8 June 2022, accessed 21 June 2022
[9] TGA Testimonials and endorsements in advertising, https://www.tga.gov.au/testimonials-and-endorsements-advertising, published 26 May 2022, accessed 21 June 2022
[10] TGA, How to demonstrate the efficacy of listed medicines is acceptable, https://www.tga.gov.au/resource/evidence-guidelines, published 21 June 2022, accessed 21 June 2022
[11] OGTR News Update – April 2022, https://www.ogtr.gov.au/sites/default/files/2022-04/newsletter_-_issue_6.pdf,
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