Included in all General Medical Registrations. 

When: Friday 2 August | 1:15pm - 2:15pm

Where: Room P11, Plaza Level, BCEC

Speaker: A/Prof. Jennifer Goralski

ACFC Vertex Lunch Symposium Synopsis

Date: Friday 2 August 2024

Time: 1:15 – 2.15pm

Title of the Symposium:  US Experience of Treating Preschool Children* with TRIKAFTA

*Aged 2–5 years

Chair: Prof. Philip Robinson, Paediatric Respiratory Physician; Director, Dept Respiratory and Sleep Medicine Royal Children’s Hospital; Research Fellow, Murdoch Children’s Research Institute, University of Melbourne, VIC. 

Speaker: A/Prof. Jennifer Goralski MD is an Associate Professor of Medicine and Paediatrics at the University of North Carolina at Chapel Hill, North Carolina, USA. A/Prof. Goralski provides clinical care to CF patients of all ages and serves as Co-Director of the UNC Adult Cystic Fibrosis Clinic. She is heavily involved in CF research and has sustained research funding from the National Institue of Health as well as the CF Foundation. She has authored over 30 manuscripts in the field of CF and conducts multicentre clinical research in CF through the Therapeutics Development Network branch of the CF Foundation. She was a lead investigator on the VX20- 445-111 trial of safety and tolerability of TRIKAFTA in preschool children ages 2–5 and was the first author on the subsequent publication in the American Journal of Respiratory and Critical Care Medicine. A/Prof. Goralski currently serves as the US national principal investigator for the VX22-445-112 study, evaluating the long-term safety and efficacy of TRIKAFTA in CF participants aged 2 years and older.

References: 1. Trikafta Approved Product Information. 2 Australian Cystic Fibrosis Data Registry, 2022

Synopsis:

On 7 Feb 2024, TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) was approved by the TGA for patients with cystic fibrosis (CF) aged 2 years and older with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.¹ Subsequently, at its March meeting, the Pharmaceutical Benefits Advisory Committee (PBAC) made a positive recommendation to extend TRIKAFTA® to this age group. With this recommendation, approximately 280 children in Australia will have access to a medicine to treat the underlying cause of their disease.²

Vertex is delighted to invite you to join an educational meeting with Associate Professor Jennifer Goralski from University of North Carolina as she shares her clinical expertise in treating preschool children aged 2 to 5 years of age with TRIKAFTA. This educational meeting will be chaired by Professor Phil Robinson.

This one-hour educational meeting will provide participants a unique opportunity to engage in peer-to-peer discussions, gain practical insights into US experience and discuss topical questions related to initiating TRIKAFTA in pre-school children.

Included in all General Medical Registrations. 

When: Saturday 3 August | 7:15am - 8:15am

Where: Room P11, Plaza Level, BCEC

ACFC Vertex Breakfast Symposium Synopsis

Date: Saturday 3 August 2024

Time: 7.15am – 8.15am

Title of the Symposium:  Evolving Challenges in CF: A Case Study Discussion

Synopsis:

The first CFTR (cystic fibrosis transmembrane conductance regulator) modulator, KALYDECO® (ivacaftor), was TGA approved in July 2013. Subsequently multiple CFTR modulators were approved: ORKAMBI® (lumacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor).^1-4

The treatment landscape for CF has undergone a significant transformation in recent years. CFTR modulators have led to improvement in health outcomes and extended lifespan in people with CF. While this is exciting for people with CF and their families, we are faced with unique and emerging challenges in CF care.^5-6

The one-hour educational meeting will be conducted in panel discussion format where CF experts from Australia, New Zealand and the United States share best practice in managing the evolving challenges in CF through case studies discussion.

This meeting provides a platform for CF clinicians and MDTs to gain practical insights from CF experts and to identify best practice sharing approaches that will guide real-world clinical settings.

CF clinicians and MDT will have the opportunity to engage in discussion on real-world case studies, Q&A and peer-to-peer exchange as our experts share their experience in managing challenging cases like mental health, carer treatment concerns and liver dysfunction in people with CF.

Panel:

Dr Jude Morton, Royal Adelaide Hospital, South Australia (Chair & speaker)
Associate Professor Jennifer Goralski, University of North Carolina, USA (Speaker)
Dr Mark O’Carroll, Auckland City Hospital, New Zealand (Speaker)

References: 1. Kalydeco Approved Product Information. 2. Orkambi Approved Product Information. 3. Symdeko Approved Product Information. 4. Trikafta Approved Product Information. 5. Australian Cystic Fibrosis Data Registry 2022 Annual Report. 6. Felipe Montiel, A et al. (2023). Expert Review of Respiratory Medicine, 17(12), 1091–1094.