January 16, 2025 Source: drugdu 34
Recently, NMPA conditionally approved the listing of China's first mesenchymal stem cell therapy, Amymetose Injection, through the priority review and approval process. Coincidentally, this is also after the FDA approved the marketing of Ryoncil, the first mesenchymal stem cell therapy, on December 18, 2024. The two drugs were approved only 15 days apart, and the indications are both for hormone-refractory acute graft resistance. Host disease.
The FDA and NMPA have successively approved the first stem cell drugs, indicating that innovative stem cell drugs have arrived.
The tortuous road
Looking back at the development and application history of Ryoncil cell drug, we can find that its road to market in the United States was extremely difficult. It took 5 and a half years from the first application in 2019 to the final approval. During this period, I received 2 CRL (application rejection) letters and was in danger of being stranded. It was not until March this year that Mesoblast finally received good news from the FDA. The FDA stated that after further consideration, it believed that the submitted phase III clinical data could support its submission of Ryoncil BLA application. According to previously disclosed clinical data, the objective response rate after 28 days of Ryoncil treatment was 70.4%, a statistically significant increase compared with the historical control rate of 45%. Among patients who received at least one infusion and were followed for 100 days, the mortality rate was 22%, while the mortality rate at day 100 was as high as 70% among patients who did not respond to steroid therapy. Finally, the FDA approved Mesoblast’s third marketing authorization application on December 18, 2024. It is worth mentioning that the product ingredient remestemcel-L has been successfully approved for marketing in Canada, New Zealand (sold under the trade name Prochymal) and Japan (sold under the trade name Temcell) as early as 2012 and 2016 respectively.
On the other hand, the road to listing of Aimimetosai has not been smooth sailing. As early as March 2013, the drug submitted an IND in China . However, due to the complexity of stem cell therapy and the stringent regulatory requirements, its development progress has been relatively slow. It was not until June 2020 that its clinical trial was announced for the first time, and it was included in the priority review and approval by CDE on June 12, 2024. The NDA was officially submitted on the 25th of the same month, and was finally approved for marketing on January 2, 2025. Although in the exploratory phase II clinical trial, the MSC group did not show a higher overall response rate (ORR) compared with the placebo group on day 28, MSCs gradually showed a therapeutic effect after an average of 2 weeks, especially Patients who completed 8 infusions and had intestinal involvement benefited significantly.
The difficult road to market for Ryoncil and Amytocer reflects, on the one hand, that both China and the United States have adopted extremely strict regulatory review systems for stem cell therapies. On the other hand, there are indeed products that have been approved in other countries. However, these products do not meet the standards of the FDA and NMPA and therefore cannot be approved, which reflects the complexity of approval and supervision in different countries.
Boom
According to Research and Markets, the global stem cell market will reach US$15.07 billion in 2023 and is expected to grow from US$17.02 billion in 2024 to US$56.15 billion in 2032, with a compound annual growth rate of 16.1% during the forecast period. In addition, data from Southern Fortune Network shows that by 2025, China’s stem cell medical market is expected to reach US$12.6 billion, with a compound annual growth rate of 19.1%.
The two approved stem cell drugs in China and the United States both focus on mesenchymal stem cells, which are a group of cells with self-renewal and multi-lineage differentiation capabilities. They are widely distributed in various tissues and organs of the human body, such as bone marrow, umbilical cord, umbilical cord blood or fat. This type of cells has the potential to differentiate into a variety of mesenchymal cells (such as osteoblasts, chondrocytes, adipocytes, etc.) or non-mesenchymal cells, and has the function of secreting cytokines. It is widely used in regenerative medicine and cell therapy. The frontier shows amazing application potential.
Globally, mesenchymal stem cells are a popular branch of stem cell therapy, and their research and application are booming. As of now, 655 mesenchymal stem cell projects around the world have entered the clinical research and development stage, far ahead of other types of stem cell therapies. Currently, there are about 10 mesenchymal stem cell therapy drugs approved for marketing around the world, and their cell sources and indications vary. Among them, there are 4 mesenchymal stem cell therapy drugs for the treatment of acute graft-versus-host disease (aGVHD). In addition to the Amimetose injection approved by China , there are also bone marrow mesenchymal stem cell drugs approved by the United States, Canada, and Japan. stem cells.
Acute graft-versus-host disease (aGVHD) is selected as the first indication for mesenchymal stem cell (MSCs) therapeutic drugs, mainly because aGVHD patients who are ineffective in hormone therapy are seriously life-threatening and have huge unmet clinical value and medical treatment. needs, and MSCs have unique immunomodulatory properties, can effectively inhibit T cell activation and inflammatory response, and theoretically have significant curative effect on aGVHD. In addition, the low immunogenicity of MSCs reduces the risk of host rejection and improves the safety of treatment. Early studies and clinical trials have initially confirmed the potential therapeutic effect of MSCs on aGVHD, providing scientific basis for further development. In
China , in addition to Amymetose, according to statistics, between January 1 and December 24, 2024, a total of 29 companies obtained new drug clinical trial (IND) applications accepted/IND approved, involving 30 drugs There are a total of 39 IND-related records for candidate drugs. This data has increased compared with last year, showing the activity and potential of
China's stem cell research and development. It is not difficult to see that among these stem cell product candidates, mesenchymal stem cells occupy a dominant position. Human umbilical cord mesenchymal stem cells, uterine blood mesenchymal stem cells, adipose mesenchymal stem cells, human amniotic membrane mesenchymal stem cells, bone marrow mesenchymal stem cells and other types have emerged one after another. Among them, human umbilical cord mesenchymal stem cells have become the most important type of research and development due to their easy availability, low immunogenicity and wide clinical application.
Currently, stem cell therapy covers hundreds of indications for almost all human body systems, such as diabetes, psoriasis, cerebral palsy, enteritis, premature ovarian failure, liver cirrhosis, systemic lupus erythematosus, COPD, osteoarthritis, rheumatoid arthritis, Male erectile dysfunction, etc.
With the continuous advancement and innovation of stem cells, the pace of research on stem cell products in China has accelerated. More and more products are entering the clinical stage, showing huge application potential and market prospects. In addition to Platinum, there are currently three stem cell products in China that have reached the Phase III clinical development stage:
1. Sibiman Bio: AlloJoin (clinical phase III)
AlloJoin is an allogeneic human fat developed by Sibiman Biotech Mesenchymal progenitor cell injection designed to treat knee osteoarthritis (KOA). On April 3, 2023, Sibiman officially announced the launch of AlloJoin’s Phase III clinical trial. In the completed randomized, double-blind, multi-center phase II clinical trial (CTR20192352), AlloJoin can significantly and continuously improve subjects’ knee joint pain and knee joint function, delay articular cartilage wear, and improve patients’ quality of life. After 96 weeks of follow-up, the efficacy remained stable. This product is not only the first innovative stem cell drug independently developed in China and approved by CDE to directly enter Phase II clinical trials, it is also the first stem cell treatment program for knee osteoarthritis in China to enter Phase III clinical trials.
2. IxCell Bio: Human Umbilical Cord Mesenchymal Stem Cell Injection (Clinical Phase III)
The human umbilical cord mesenchymal stem cell (IxCell hUC-MSC-O) injection developed by Aisar Bio is used to treat knee osteoarthritis. It has now entered clinical phase III. Previously, phase II clinical studies have successfully completed nearly 200 cases, demonstrating its good efficacy. This "off-the-shelf" umbilical cord-derived MSC drug candidate has the ability to efficiently differentiate into a variety of tissue cells such as adipocytes, osteoblasts, and chondrocytes. It also has low immunogenicity and does not require histocompatibility matching for clinical treatment. , to avoid medical ethics issues.
3. Madison Regenerative Medicine: Premetose Injection (Clinical Phase III)
Premetose Injection is a bone marrow-derived original mesenchymal stem cell preparation developed by Madison Regenerative Medicine. It is closely related to Platinum’s excellent Amy Metocel injection is similar and is mainly used to treat graft-versus-host disease (GVHD). The Phase III clinical trial of this product was registered on July 5, 2023 (CTR20230686), and is planned to be conducted in multiple hospitals across the country as a multi-center clinical study, with approximately 100 patients expected to be recruited. Currently, the trial is still in the recruitment phase.
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