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

 

Leveraging Expedited Regulatory Pathways to Optimize Drug Development

A Pharmafocus webinar

Join us on Thursday 24th September 2020, 15:00 BST | 16:00 CEST | 10:00 EDT, when Dr Frank Casty, Judith Plon and Professor Steffen Thirstrup will guide you through the expedited regulatory pathways available in the EU and US and discuss how to effectively leverage these to accelerate and optimize your drug development program.

A timely market introduction is a critical component of any drug development strategy – not only from a commercial standpoint, where an earlier market entry can lead to a competitive advantage, but also for patients awaiting better treatment options.

Regulators worldwide have developed specialized pathways to expedite review and approval of new drugs. Navigating these pathways can be a daunting task for sponsors.

Partnering with experienced regulators both in the US and EU is critical to achieving expedited reviews since getting the strategy right, developing effective documentation, and preparing for regulatory interactions can make the difference between a standard regulatory review and a successful expedited review

Our panel will discuss:

  • Which pathways are available
  • When to apply for specific designations
  • What data are required and what to expect if the designation is granted
  • Best practice to create a successful strategy

Click here to book your place today!


About the Panel

Dr Frank Casty: Senior Clinical Regulatory Advisor, NDA Group

Frank works with small and large companies across several therapeutic areas including cardiovascular, respiratory, inflammation, oncology and rare diseases.  He has led numerous FDA interactions, IND and NDA filings as well as presented at successful FDA Advisory Committee Meetings.

Before joining NDA, Frank worked for more than 25 years in small and large pharmaceutical companies leading Global Clinical Research and Medical Affairs organizations as well as serving as technical consultant for Business Development activities.

Judith Plon: Principal Consultant, NDA Group

Judy works with small and large companies across multiple therapeutic areas. She serves as a US Agent for clients leading the submission of their INDs, NDAs, and special designation requests (i.e. Orphan Drug Designations).

Prior to joining NDA Judy spent over 25 years in the Biopharma Industry, primarily working in the discipline of regulatory affairs. She held positions of increasing responsibilities and last served as a Vice President of Global Regulatory Affairs.

Professor Steffen Thirstrup: Director NDA Advisory Board, NDA Group

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.

Prior to joining NDA in 2013, Steffen was employed as the head of Division for Medicines Assessment and Clinical Trials at the Danish Health and Medicines Authority where he also acted as Danish member of the Committee for Human Medicinal Products (CHMP) at the EMA.