Advanced Therapies in Wound Management: What Needs to be Considered for an Integrated Development Strategy
by Diane Seimetz
The great potential of advanced therapies in wound management was recently demonstrated by a case study, where the entire human epidermis of a boy with junctional epidermolysis bullosa (JEB) was regenerated using genetically modified autologous keratinocytes [1].
Despite, the game changing potential of advanced therapies, the success rate at the time of marketing authorization application is rather poor [2]. Over the past 11 years, only 12 advanced therapies received a positive opinion for authorization in the EU. None of these products were indicated for wound management or skin related diseases.
Very few new drugs for wound management became available during the past two decades. Only 2 products, i.e. Regranex (beclapermin) and Episalvan (birch bark extract) were centrally approved for wound healing in the EU. In the meantime, the Regranex marketing authorization was even withdrawn due to commercial reasons. Clinical development in wound related indications is notoriously difficult with numerous failed pivotal studies despite encouraging signals during earlier clinical development stages [3].
In addition, companies developing advanced therapies are often small and medium sized enterprises, who have typically less experience in drug development compared to large size pharmaceutical companies.
Thus, companies developing innovative advanced therapies for wound management are facing a combination of three challenges, i.e. the advanced therapy challenge, the indication challenge and the SME challenge.
Therefore an integrated development and regulatory strategy is of utmost importance to realize the potential of advanced therapies in wound management.
An overview of important criteria that need to be considered at each transition stage from R&D to manufacturing trough nonclinical and clinical development up to authorization are provided in Figure 1.
For further details, please refer to the article written by Dr. Diane Seimetz and Dr. Annegret Vaggelas in the HERE.
Interested to learn more about how we can support setting up your integrated development and regulatory strategy? Don’t hesitate, get in contact with us!
Dr. Diane Seimetz was invited to present on Advanced Therapy Medicinal Products in Wound Management at the European Wound Management Association being held on 9 to 11 May 2018 in Krakow.
- Hirsch T, Rothoeft T, Teig N, Bauer JW, Pellegrini G, De Rosa L, et al. Regeneration of the entire human epidermis using transgenic stem cells. Nature. 2017;551(7680):327-32.
- Seimetz D. ATMPs: How to successfully master Challenges and foster the Regulatory Success rate? Pharmazeutische Medizin. 2016;(18) 3.
- Maderal AD, Vivas AC, Eaglstein WH, Kirsner RS. The FDA and designing clinical trials for chronic cutaneous ulcers. Seminars in Cell and Developmental Biology. 2012;23(9), 993–999.
