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Australian Public Assessment Report for Tafamidis and Tafamidis Meglumine

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Australian Public Assessment Report for Tafamidis and Tafamidis Meglumine Australian Public Assessment Report for Tafamidis and Tafamidis meglumine Proprietary Product Name: Vyndamax and Vyndaqel Sponsor: Pfizer Australia Pty Ltd September 2020 Therapeutic Goods Administration About the Therapeutic Goods Administration (TGA) • The Therapeutic Goods Administration (TGA) is part of the Australian Government Department of Health and is responsible for regulating medicines and medical devices. • The TGA administers the Therapeutic Goods Act 1989 (the Act), applying a risk management approach designed to ensure therapeutic goods supplied in Australia meet acceptable standards of quality, safety and efficacy (performance) when necessary. • The work of the TGA is based on applying scientific and clinical expertise to decision- making, to ensure that the benefits to consumers outweigh any risks associated with the use of medicines and medical devices. • The TGA relies on the public, healthcare professionals and industry to report problems with medicines or medical devices. TGA investigates reports received by it to determine any necessary regulatory action. • To report a problem with a medicine or medical device, please see the information on the TGA website <https://www.tga.gov.au>. About AusPARs • An Australian Public Assessment Report (AusPAR) provides information about the evaluation of a prescription medicine and the considerations that led the TGA to approve or not approve a prescription medicine submission. • AusPARs are prepared and published by the TGA. • An AusPAR is prepared for submissions that relate to new chemical entities, generic medicines, major variations and extensions of indications. • An AusPAR is a static document; it provides information that relates to a submission at a particular point in time. • A new AusPAR will be developed to reflect changes to indications and/or major variations to a prescription medicine subject to evaluation by the TGA. Copyright © Commonwealth of Australia 2020 This work is copyright. You may reproduce the whole or part of this work in unaltered form for your own personal use or, if you are part of an organisation, for internal use within your organisation, but only if you or your organisation do not use the reproduction for any commercial purpose and retain this copyright notice and all disclaimer notices as part of that reproduction. Apart from rights to use as permitted by the Copyright Act 1968 or allowed by this copyright notice, all other rights are reserved and you are not allowed to reproduce the whole or any part of this work in any way (electronic or otherwise) without first being given specific written permission from the Commonwealth to do so. Requests and inquiries concerning reproduction and rights are to be sent to the TGA Copyright Officer, Therapeutic Goods Administration, PO Box 100, Woden ACT 2606 or emailed to <[email protected]>. AusPAR - VYNDAMAX and VYNDAQEL - tafamidis and tafamidis meglumine - Pfizer Australia Pty Ltd - Page 2 of 40 PM-2019-00391-1-3 and PM-2019-01399-1-3 FINAL 3 September 2020 Therapeutic Goods Administration Contents Common abbreviations _____________________________________________________ 4 I. Introduction to product submission ____________________________________ 7 Submission details ____________________________________________________________________ 7 Product background __________________________________________________________________ 9 Regulatory status ____________________________________________________________________ 10 Product Information_________________________________________________________________ 11 II. Registration timeline __________________________________________________ 11 III. Submission overview and risk/benefit assessment ______________ 12 Quality ________________________________________________________________________________ 12 Nonclinical ___________________________________________________________________________ 13 Clinical ________________________________________________________________________________ 14 Risk management plan ______________________________________________________________ 27 Risk-benefit analysis ________________________________________________________________ 29 Outcome ______________________________________________________________________________ 38 Attachment 1 and 2. Product Information _____________________________ 39 AusPAR - VYNDAMAX and VYNDAQEL - tafamidis and tafamidis meglumine - Pfizer Australia Pty Ltd - Page 3 of 40 PM-2019-00391-1-3 and PM-2019-01399-1-3 FINAL 3 September 2020 Therapeutic Goods Administration Common abbreviations Abbreviation Meaning 6MWT 6 minute walk test ACM Advisory Committee on Medicines ACSS Australia-Canada-Singapore-Switzerland AE Adverse event ARTG Australian Register of Therapeutic Goods ATTR Transthyretin amyloid ATTR-CM Transthyretin amyloid cardiomyopathy ATTR-PN Transthyretin amyloid polyneuropathy AUC Area under the concentration-time curve BCRP Breast cancer resistant protein CER Clinical evaluation report CHMP Committee for Medicinal Products for Human Use (European Union) CI Confidence interval CL/F Apparent clearance CM Cardiomyopathy Cmax Maximum observed plasma concentration CMI Consumer Medicines Information CV Cardiovascular CYP3A4 Cytochrome P450, family 3, subfamily A, polypeptide 4 EAIR Exposure-adjusted incidence rate EMA European Medicines Agency (European Union) Emax Maximal response AusPAR - VYNDAMAX and VYNDAQEL - tafamidis and tafamidis meglumine - Pfizer Australia Pty Ltd - Page 4 of 40 PM-2019-00391-1-3 and PM-2019-01399-1-3 FINAL 3 September 2020 Therapeutic Goods Administration Abbreviation Meaning EU European Union FDA Food and Drug Administration (United States) GVP Good Pharmacovigilance Practice(s) ICH International Council for Harmonisation IRD Incidence rate difference IRR Incidence rate ratio KCCQ-OS Kansas City Cardiomyopathy Questionnaire – overall summary LS Least squares LVEF Left ventricular ejection fraction MRI Magnetic resonance imaging NMT Not more than NT-proBNP N-terminal prohormone of brain natriuretic peptide NYHA New York Heart Association OAT Organic anion transporter PBRER Periodic benefit-risk evaluation report PD Pharmacodynamics(s) PF-06291826 Drug development/sponsor code for tafamidis PI Product Information PK Pharmacokinetic(s) PK-PD Pharmacokinetic-pharmacodynamic PSUR Periodic safety update report PT Preferred Term SAE Serious adverse event SY Subject years of exposure AusPAR - VYNDAMAX and VYNDAQEL - tafamidis and tafamidis meglumine - Pfizer Australia Pty Ltd - Page 5 of 40 PM-2019-00391-1-3 and PM-2019-01399-1-3 FINAL 3 September 2020 Therapeutic Goods Administration Abbreviation Meaning T4 Thyroxine TEAE Treatment-emergent adverse event(s) TESPO Tafamidis Enhanced Surveillance Pregnancy Outcomes Tmax Time to reach maximum plasma concentration after drug administration TSH Thyroid-stimulating hormone TTR Transthyretin TTRR Tafamidis: transthyretin ratio ULN Upper limit of normal US United States UTI Urinary tract infection vs Versus AusPAR - VYNDAMAX and VYNDAQEL - tafamidis and tafamidis meglumine - Pfizer Australia Pty Ltd - Page 6 of 40 PM-2019-00391-1-3 and PM-2019-01399-1-3 FINAL 3 September 2020 Therapeutic Goods Administration I. Introduction to product submission Submission details Vyndamax (Submission PM-2019-00391-1-3) Type of submission: New chemical entity Decision: Approved Date of decision: 13 March 2020 Date of entry onto ARTG: 16 March 2020 ARTG number: 314813 Yes. This product will remain in the scheme for 5 years, Black Triangle Scheme:1 starting on the date the product is first supplied in Australia. Active ingredient: Tafamidis Product name: Vyndamax Sponsor’s name and address: Pfizer Australia Pty Ltd Level 17, 151 Clarence Street, Sydney NSW 2000 Dose form: Soft capsule Strength: 61 mg Container: Blister pack Pack size: 30 Approved therapeutic use: Vyndamax is indicated for the treatment of adult patients with wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM). Route of administration: Oral Dosage: Treatment should be initiated and remain under the supervision of a physician knowledgeable in the management of patients with transthyretin amyloid cardiomyopathy 1 The Black Triangle Scheme provides a simple means for practitioners and patients to identify certain types of new prescription medicines, including those being used in new ways and to encourage the reporting of adverse events associated with their use. The Black Triangle does not denote that there are known safety problems, just that the TGA is encouraging adverse event reporting to help us build up the full picture of a medicine's safety profile. AusPAR - VYNDAMAX and VYNDAQEL - tafamidis and tafamidis meglumine - Pfizer Australia Pty Ltd - Page 7 of 40 PM-2019-00391-1-3 and PM-2019-01399-1-3 FINAL 3 September 2020 Therapeutic Goods Administration (ATTR-CM). The recommended dose of Vyndamax is 61 mg tafamidis orally once daily (see Section 5.1 Pharmacodynamic properties of the Product Information). A single 61 mg Vyndamax (tafamidis) capsule is bioequivalent to 80 mg Vyndaqel (tafamidis meglumine) (administered as four 20 mg Vyndaqel capsules) and is not interchangeable on a per mg basis (see Section 5.1 Pharmacodynamic properties; and Section 5.2 Pharmacokinetic properties, of the Product Information). No dose ranging studies have been undertaken. For further information regarding dosage, refer to the Product Information. Vyndaqel (Submission PM-2019-01399-1-3) Type of submission: New chemical entity Decision: Approved Date of decision: 13 March 2020
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