Advanced Therapies Week Countdown: 2022 in Approvals and a Patient-Centric Approach

Anna Osborne
6 January 2023
Cell Therapy
Clinical Trials
Gene Therapy
Patient Access & Engagement
The countdown to Phacilitate’s Advanced Therapies week 2023 is well and truly on! This year the FDA will be joining us to discuss issues of regulation and approval in the context of this patient-centric industry.

It has been a record-breaking year for advanced therapies with more cell and gene therapies approved than ever before and lots of incredible patient stories shared, and there will be lots to celebrate when we come together at the Miami Beach Convention Centre!

This year our agenda is lucky enough to feature presentations from some hugely influential patient advocates alongside presentations from regulatory experts from the FDA. If there is one thing that underpins our industry, it is a focus on patients, for whom these advanced therapies often offer a last chance of hope.

Something our audience, myself included, note about Advanced Therapies Week every year is just how the poignant stories from patients and patient advocates really bring home why everyone in this area is doing what they are doing. The patient is therefore at the center of everything we do at Advanced Therapies Week.

The question is, how can we overcome obstacles of market access and approval to allow for these products to reach patients?

Before we take a look at some sessions combining regulatory insights with patient needs, let’s take a brief look back at the six new cell and gene therapy approvals we saw in 2022.

Approval Successes of 2022
  • Carvykti (CAR-T) – Legend Biotech and Janssen, FDA US (Feb) and EU (May) for r/r multiple myeloma.
  • Upstaza (Gene Therapy) – PTC Therapeutics, EU (Jul), for Aromatic L-amino acid decarboxylase (AADC) deficiency
  • Roctavian (Gene Therapy) – BioMarin Pharmaceutical, EU (Aug), for Haemophilia A
  • Eli-cell (Gene Therapy) – bluebird bio, FDA US (Sept), for Early active cerebral adrenoleukodystrophy (CALD)
  • Hemgenix (Gene Therapy) – CSL/UniQure, FDA US (Nov), for Haemophilia B
  • Ebvallo (Cell Therapy) – Atara Biotheraputics, EU (Dec), for Epstein-Barr virus-associated post-transplant lymphoproliferative disorder (EBV + PTLD)

And there is a lot to look forward to as we enter 2023, with 13 CGTs expected to be considered by the FDA, and 3 by the EC, for approval in 2023, according to Alliance for Regenerative Medicine. It’s clear the relationship between patient advocates and big biopharma is more important now, than ever.

Find out more. Join us at Advanced Therapies Week 2023 >>

Sessions to Look Out For

1.Working with Patient Advocacy Organizations to Inform Development

Chaired by Sharon Hesterlee, Chief Research Officer at the Muscular Dystrophy Association USA, this Patient and Clinical session features presentations from Tom Whitehead, Co-Founder, Emily Whitehead Foundation, Tessa Rowe, User Experience and Human Factors Engineering, Tensentric, Gwen Nichols, Chief Medical Officer Leukemia and Lymphoma Society (LLS), and Anne Rowzee, Associate Director for Policy, OTAT, FDA.

Gwen Nichols’ presentation ‘Supporting Blood Cancer Patients: LLS’s Holistic 360◦ Approach’. The LLS works to support patients and families through their blood cancer journeys. Through patient advocacy, research, education, and financial support LLS are also able to work with biopharma companies to help them understand and target the unique needs of patients with blood cancer. This partnership between patient-focused societies and biopharma companies is essential – the patient must be at the start and end of every advanced therapeutics journey.

Gwen will share the ways in which LLS and biopharma have worked together successfully in order to advance the next generations of immunotherapy and how taking a 360 approach is beneficial to all involved.

In light of the patient-centric stories shared in this session, the final presentation ‘Incorporating Patient Perspectives at FDA, a Continuing Evolution’ by Anne Rowzee, offers a nice comparison and insight into how the FDA incorporates patient perspectives sharing the way in which the FDA gathers patient input.

With a chance for open audience discussion in the closing panel Q&A this is set to be a very thought-provoking session not to be missed.

 You can join this session ‘Working With Patient Advocacy Organizations to Inform Development’ at Advanced Therapies Week, Thursday January, 19 at 11am.

2. How Do We Best Serve Rare and Small Patient Populations

Chaired by Christian Chabannon, Head of Center for Cell Therapy, Institut Paoli-Calmettes, Marseille, EBMT & GoCART this session features an opening presentation from Wilson Bryan, Director, Office of Tissues and Advanced Therapies, FDA  – ‘Gene Therapy for Rare Diseases: Can We Go Faster and Still Get It Right?’.

In this presentation, Wilson will explore how scientific advances have made gene therapy a reality for a small number of rare diseases, as we can see in the current cell and gene therapy FDA approvals.

It is important to celebrate the regulatory success we have seen so far, but in the same breath, we all know there is a very long way to go. The presentation will also go on to cover the need for rigorous natural history studies and clinical trials for the safe and efficient development of new gene therapies.

The session will also see presentations from Brad Carlin, Senior Advisor, Statistics and Data Science, PharmaLex, US, Allyson Berent CSO, Foundation for Angelman Syndrome Theraputics (FAST), and, Sharon Hesterlee, CRO, Muscular Dystrophy Association, USA.

As both a parent of a child with a genetic disorder and CSO of FAST, Allyson Berent will share her unique perspective in the presentation ‘A Parents’ Journey Through Drug Development”: How Angelman Syndrome is Making the Impossible Possible’. Allyson will detail the amazing work the foundation has made to see their vision for a transformative treatment for Angelman Syndrome become a reality, as well as a day in her life as she navigates the world of rare diseases as a parent.

You can join this session ‘How Do We Best Serve Rare and Small Patient Populations’ at Advanced Therapies Week, Friday January, 20 at 11am.


Join us at Advanced Therapies Week >>