2017 marks 20 years since the first regulatory approval of cell-based therapeutic product on a market. In 1997 Carticel (manufactured by Genzyme Corp.) was approved by FDA for US market. One year before Carticel approval, 2 cell-based products were launched and marketed for the first time in Europe (Italy) – Laserskin and Hyalograft by Fidia Advanced Biopolymers. I was curious to learn about other approved and marketed cell-based products worldwide in the last 20 years. I also wanted to learn what happen to the first products, marketed 15-20 years ago. It took me almost a year to investigate this topic, collecting bits of information from public sources and conferences. Today, I’m sharing some of my data analysis.
I searched published literature, company press releases, mass media coverage, books (via Google books), patents, presentations from conferences and meetings, information from regulatory agencies. Also, I’ve learned some unique information from multiple conferences and from personal communications.
- the product must contain alive cells and used for therapeutic purpose;
- the product must be marketed and/ or approved by regulatory agency;
- cord blood products for homologous use, obtained BLA as FDA requirement in US;
- traceable public information is available.
- absence of traceable public information when and where product was marketed;
- tissues for transplantation without brand name (not a product);
- More than 3000 therapeutic products marketed in Japan, as Specific Processed Cellular Products under the Act on the Safety of regenerative Medicine (ASRM) – Class III Regenerative Medicine products (not regulated under the PMDA). In this case, individual institutions (hospitals) submit a “Plan to Provide Regenerative Medicine” and seek for approval from Japanese Ministry of Health, Labour and Welfare (MHLW). Information about 3 ASRM products was publicly available and searchable. These 3 products were included in the dataset as an exception.
For historical analysis I was trying to capture the earliest available data. Some early approvals and self-launches happened before defining regulatory framework for cell/ tissue products. So, some cell-based products were approved as devices. The following data were captured: name of the product, name of the manufacturer, year of approval and/ or when it was launched on the market, where it was marketed, name of regulatory agency (if it was approved), indication, cell type, traceable changes (off market, manufacturer went for bankruptcy…). The whole dataset is available on Cell Trials Data.
(1) As of March 2017, I was able to identify 90 marketed cell-based products. There were mentions of few products, without traceable information of when it was launched (for example, Cartogen in Australia). 61% (55/90) of marketed products were approved by regulatory agencies; 12% (11/90) were self-launched with manufacturing license, issued by governmental agency; 27% (24/90) were self-launched without information about manufacturing license.
How to cite this figure: Bersenev, Alexey (2017): Self-launched versus approved cell-based products, marketed worldwide. figshare. https://doi.org/10.6084/m9.figshare.4829452
(2) Most of cell-based products were marketed for 3 major groups of indications: skin defects (31%), cartilage repair (24%) and oncology (19%).
By the time of writing this post, at least 12 products (13%) were off market. This number is underestimated, since there was no public information available about current status of many products, marketed earlier. At least 5 manufacturers went to bankruptcy, some were acquired or outlicensed products to other companies.
(3) Since 1997, there were 58 regulatory approvals of 55 cell-based products by 13 different jurisdictions. 3 products were approved by more than one jurisdiction (Provenge, Prochymal and MACI).
How to cite this figure: Bersenev, Alexey (2017): Regulatory approvals of cell-based therapeutic products by jurisdiction. figshare. https://doi.org/10.6084/m9.figshare.4829182.v1
(4) Historical trend for approvals is slowly going up with some volatility. Number of approvals per year was ranging from 0 to 8.
How to cite this figure: Bersenev, Alexey (2017): Historical trend in regulatory approvals of cell-based therapeutic products worldwide. figshare.
How to cite this post:
Bersenev Alexey. Historical analysis of cell-based therapeutic products marketed and approved worldwide. CellTrials blog. April 7, 2017. Available: http://celltrials.info/2017/04/07/marketed-approved
PS: I’d like to thank Colin Lee Novick for clarifying information about regulation in Japan.