Björn Carlsson joins the NDA team!

We are excited to welcome Björn Carlsson. Björn comes from a comprehensive scientific and regulatory background and has extensive experience in ATMPs. We have no doubt that he will be a valuable asset to our team.

In this interview we take the opportunity to get to know him a little better.


Welcome Björn Carlsson, tell us a bit about your background.

Thank you very much. I started out as a research scientist at Uppsala University in Sweden where I focused on bench-to-bedside adoptive T-cell therapy.  Later, I became an associate professor in industrial experimental clinical immunology.

In 2008 I started working for the Swedish Medical Products Agency as a non-clinical assessor, evaluating everything in terms of the development of new drugs. It covered the whole process from first-in-human trials to market authorizations and included every kind of pharmaceutical; chemicals, biologics, biosimilars, and ATMPs.

Thanks to my in-depth scientific knowledge of ATMPs, I was appointed the Swedish substitute delegate in the committee for advanced therapies at the European Medicines Agency. This helped me gain international scientific and regulatory knowledge of advanced therapies. For the last four years, I have used my experiences within the field of ATMPs as a Horizon 2020 evaluator for the EU commission.

That is quite an interesting background. How do you see your experience helping NDA’s clients?

I believe that I can contribute in many different areas of the drug development process. I have a lot of experience in non-clinical development as we as extensive experience in how the regulatory system works.

In addition, my long experience with ATMPs would be of great interest, especially when it comes to the non-clinical packages, which are tremendously specialized and different from traditional drugs, including biotech drugs. There is a great need to have expertise in this area, from progress, via CMC, into clinical development.

The most challenging part of the ATMP non-clinical development is to generate relevant data in order to make a comprehensive risk assessment. Without such evaluation, it is very difficult to get approval by the regulatory agencies, especially within non-life-threatening indications. I do believe this is another area I can contribute to.

What do you see happening in the Nordic Life Science landscape, anything that gets you excited?

There are a lot more initiatives these days compared to how it used to be, people are much more organized now. There has also been a widespread recognition that there is legislation around ATMP products to which we need to adhere to.

In the early days of the legislation for advanced therapies, the community did not really like to be regulated. Many of the developers were under the impression that the products they were developing were for transplantation or standard of care, rather than an experimental drug. However, due to local regulatory agencies and financial contributors, this has changed a lot. I would say that one of NDA’s strengths is that we are well equipped to deal with these regulations

What in your personality adds an extra asset to your professional persona?

I would say that I am a very diplomatic person. I adapt easily to new people and new ways to work. I prefer working with other people and want to be a contributing factor to the collective intelligence. I am a social kind of guy who takes pride in being available and takes time to talk to people.

What are your expectations of working at NDA?

What made me transfer from the regulatory agency to the consulting business is the fact that now I will be working with a team that helps develops drugs. This is a sharp contrast to assessing it from the agency’s point of view, where you were just looking at what has been done. Now I can influence the process as it unfolds, helping to make it right from the start, and perhaps saving the developers some headaches.

Are you looking for deep regulatory and strategic knowledge to support your drug development program? Email us at info@ndareg.com to get in touch.

How to Keep up With The Fast-Evolving World of ATMPs

Join us on Thursday 19th November 2020, 15:00 GMT | 16:00 CET | 10:00 EST, when Dr Paula Salmikangas and Professor Steffen Thirstrup discuss how to keep up with the Fast-Evolving World of ATMPs.

The development of novel advanced therapy medicinal products (ATMPs) is moving fast, as witnessed by the growing number of products appearing in clinical trials. Investments and ATMP developers are also increasing worldwide. Innovative technologies like gene editing have enhanced the design of products to better target the indications – for example, by allowing the move from autologous products to allogeneic, off-the-shelf products.

Advances in virus vector production systems have greatly improved the safety profiles of current gene therapy products. However, the design and engineering of ATMPs are becoming more and more complex, which brings new and possibly unknown risks and regulatory concerns. The availability of non-clinical models for safety testing is limited for these products and it may be that some risks are identified only through human exposure; this requires careful planning of the clinical studies and robust strategies for risk mitigation and long-term pharmacovigilance.

The challenges of persistency and immunogenicity for some products, such as adeno-associated viruses (AAVs), are currently hampering their long-term effectiveness and the overall clinical outcomes. Outstanding efficacy results for approved products like Kymriah®, Yescarta®, and Zolgensma® are strongly encouraging the field towards curative treatments, but the risks related to novel technologies should remain manageable and well-controlled to ensure real benefits for patients.

The key discussion points of this webinar will include:

  • An overview of the current status of approved ATMPs and those in clinical trials worldwide
  • Recognition of novel manufacturing technologies and their perceived risks
  • Understanding the pros and cons of non-clinical studies, including risk identification and mitigation strategies
  • An update on recent clinical results and new trial designs, including histology agnostic trials

 

Click to book your place today

If you require further information, please do not hesitate to contact us.

 

We are expecting this webinar to generate a lot of audience interest, so please send in your questions now to ensure the speakers have time to answer them in the Q&A: email our moderator at conor@pharmafile.com. If your question is for a specific presenter, please include this in your email.


Speakers:

Dr. Paula Salmikangas, Director of Biopharmaceuticals and ATMPs, NDA Group

Paula is the Director of Biopharmaceuticals and ATMPs and has been at NDA since 2017. Her main areas of expertise are biological medicinal products, especially advanced therapy medicinal products and the CMC aspects of biopharmaceuticals. As a former EU regulator, Paula was actively involved in establishing the regulatory guidelines for ATMPs and applies her unique experience to provide companies with technical & strategic advice for their drug development programs.

 

Professor Steffen Thirstrup, Director NDA Advisory Board, NDA Group

Steffen has been with NDA since 2013 and excels at advising companies on their development strategies to meet the expectations of regulatory agencies around the world. He applies his skills to a broad range of therapeutic areas and has successfully helped numerous clients interact with regulatory agencies throughout the stages of development and during regulatory review.

 

Moderated by Conor Kavanagh, Journalist, and Editorial Assistant, Pharmafocus

 

Integrated product development for ATMPs

By: Claes Buxfeldt and Dr Paula Salminkangas

In this article, published in the September issue of MedNous, Claes and Paula discuss why an integrated product development strategy for ATMPs is essential to meet both regulatory and HTA requirements.


Advanced Therapy Medicinal Products (ATMPs), which include cell and gene therapies and tissue engineered products, are a group of innovative products targeting diseases and conditions for which there are few, if any, effective treatments. The success of the first CD19 chimeric antigen receptor T cell (CAR T) products Kymriah and Yescarta against B-cell malignancies has raised the awareness of the high potential of ATMPs, but also shown the several challenges relating to their clinical use1. One of these challenges is the high prices asked by the manufacturers of these products, which are not always supported by national pricing and reimbursement bodies2.

In such cases, the discrepancy between a regulatory approval and a negative decision from a health technology assessment (HTA) body has raised concerns and questions from industry about how to ensure that an approved product also gets to the market and to patients. Many jurisdictions have created early access schemes and ways to communicate with regulatory and HTA bodies early on to ensure successful outcomes of both reviews3. However, the ATMP industry is facing challenges in both aspects.

The development of ATMPs has substantially increased with a focus on clinical trials in recent years. Several cell and gene therapy products have been authorised worldwide, most recently the CAR T product Tecartus from Kite Pharma Inc.4 in the US and Zolgensma for spinal muscular atrophy (SMA)5 in the EU.

Today there are more than 980 developers globally, the majority (78%) of whom are in North America and Europe.6 Over 1,000 clinical trials were underway worldwide at the end of 2019, two-thirds of which (64%) were in Phases 2 and 3. There has been a clear shift from early to late phase trials, as only four years earlier the majority of trials (> 90%) were in Phases 1 and 27. Since beginning of 2015, the overall number of ATMP clinical trials and ATMPs in Phase 3 has doubled, suggesting multiple new ATMPs will be approaching the marketing authorisation application stage in the next few years.

Today, the focus of ATMP development is heavily in gene therapy and genetically modified cells which constitute three-quarters of products in clinical trials. This is most probably due to the fast development of novel vectors and technologies, including genome editing. In addition, a lot of safety data has accumulated for certain gene therapy approaches (e.g. adeno associated virus vectors, AAV and lentivirus vectors, LVV), which reduces the regulatory burden before first clinical trials.

From an indication perspective, the majority of ATMP clinical trials (657/1066, 62%) 6 are in oncology, including leukaemia, lymphoma, and solid tumors, which may be explained by the great interest towards novel immunotherapies using genetically modified cells. In 2019, genome edited cells using the CRISPR/Cas9 approach proceeded to clinical trials both in the US and the EU8 and the first results from a trial studying an induced pluripotent stem (iPS) cell-derived product were reported in the EU 9.

Click to the full article
 

Contact us to learn more about how our integrated team can provide advice and support into your drug development plan.

 


References :

  1. Mohty M., Gautier J., Malard F., et al. CD19 chimeric antigen receptor-T cells in B-cell leukemia and lymphoma: current status and perspectives. Leukemia 2019, 33: 2767–2778
  2. Jönsson, B., Hampson, G., Michaels, J., Towse, A., Graf von der Schulenburg, JM. and Wong, O. Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare. Eur. J. Health Econ. 2019, 20(3): 427–438
  3. Jörgensen, J. and Kefalas, P. Reimbursement of licensed cell and gene therapies across the major European healthcare markets. J Mark Access Health Policy 2015, 3: 10.3402/jmahp.v3.29321.
  4. FDA approved Cellular and Gene Therapy Products, https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products
  5. EU public assessment report for Zolgensma https://www.ema.europa.eu/en/medicines/human/EPAR/zolgensma
  6. Alliance for Regenerative Medicine: 2019 Regenerative Medicine Sector Report, available from https://alliancerm.org/sector-report/2019-annual-report
  7. 7.Alliance for Regenerative Medicine: Q1/2015 Regenerative Medicine Sector Report, available from https://alliancerm.org/wp-content/uploads/2018/04/ARM_Q12015_Data_Report_Web_Version.pdf
  8. CRISPR Therapeutics and Vertex Announce Progress in Clinical Development Programs for the Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001, available from https://investors.vrtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-progress-clinical
  9.  Cynata Completes Clinical Study Report for Phase 1 Trial of CYP-001 in GvHD, available from https://www.globenewswire.com/news-release/2018/12/18/1668688/0/en/Cynata-Completes-Clinical-Study-Report-for-Phase-1-Trial-of-CYP-001-in-GvHD.html

 

 

Optimising the path to market for ATMPs

A two part webinar series

Together with Cirio Advokatbyrå would like to invite you to our two-part interactive webinar series, Optimising the path to market for ATMPs.

ATMPs, Advanced Therapy Medicinal Products, are an increasing and diverse group of innovative products. They are complex  products with unique development challenges, and several have shown very promising efficacy results in treating highly debilitating diseases and conditions. However, the prices of the first approved ATMPs have been high and not always supported by the national pricing or reimbursement bodies or other payers.

This interactive webinar series will focus on the challenges of ATMP development and market access with case examples of approved products. The option of an integrated product development to cover both regulatory and HTA expectations will be presented, together with information on regulatory, HTA and market access support available for ATMP developers.

Session 1: Integrated Product Development for ATMPs to meet Regulatory and HTA requirements

Date: 22nd September

Time: 11am – 12pm CET

Session 2: Models for realizing ATMP- value

Date: 29th September

Time: 11am – 12pm CET

Our panelists are available to answer questions at the end of each session. You can ask your questions during the webinar or send them in advance to denise.stromquist@ndareg.com


Click here to book your place today!



Panelists 

Paula Salmikangas: Director of Biopharmaceuticals and ATMP, NDA Group

Paula joined NDA in 2017 from her position as a Research Professor at the Finnish Medicines Agency (2003-2017). She has served as a member of the EMA Committee for Advanced Therapies (CAT) from 2009 to 2017 and as the Chair of the CAT 2014-2017. She has also been the Chair of EMA CPWP and a member of the BWP. Her main areas of expertise are biological medicinal products, especially advanced therapy medicinal products and CMC aspects of biopharmaceuticals.

Steffen Thirstrup: Director NDA Advisory Board, NDA Group

Steffen joined NDA in 2013 from his position as head the Division for Medicines Assessment and Clinical Trials at the Danish Health and Medicines Authority where he alos acted as Danish member of the Committee for Human Medicinal Products (CHMP) at the EMA. Steffen excels at advising companies on their development strategies to meet expectations of regulatory agencies around the world. He applies his skills to a broad range of therapeutic areas and have successfully helped numerous clients interact with regulatory agencies throughout the stages of development and during regulatory review.

Anders Burén: Senior Counsel, Cirio Advokatbyrå

Anders specialises in strategic collaborations in the pharmaceutical industry and in regulatory law. He advises on Swedish and international aspects of agreements of all kinds governing research, development and commercialisation in the pharmaceutical industry and on product approvals, marketing and other regulatory considerations. Anders has previous experience as Assistant General Counsel for the AstraZeneca Group’s transaction lawyers based outside the US. Before that he was Assistant General Counsel for the Group’s lawyers based in Sweden.

Per Hedman:  Partner, Cirio Advokatbyrå

Per Hedman is head of Cirio’s Life Science, Health and Food practice – the Biological team. Per advises companies in all aspects of their business and legal affairs, including regulatory advice, R&D arrangements (such as non-disclosure, material transfer, clinical trial and joint development agreements), commercial relationships (such as manufacturing, supply and distribution agreements), M&A (such as product acquisitions and spin-outs, domestic and cross border), capital-raising transactions (debt and equity), and IPO:s and other capital market transactions.


To learn more about NDA’s ATMP capabilites click here