INSTITUTION PROFILE / iPS CELLS & REGENERATIVE MEDICINE
PMDA
The agency that does the hands-on review of Japan's regenerative medicines. The Minister decides approval
The Pharmaceuticals and Medical Devices Agency (PMDA) is an incorporated administrative agency supervised by the Ministry of Health, Labour and Welfare (MHLW). For regenerative medicine products, including those made from iPS cells, it handles consultations, review and inspection of manufacturing sites. The decision to approve, however, is made by the Minister of Health, Labour and Welfare, and research grants come from programmes run by AMED, the Japan Agency for Medical Research and Development. The two iPS cell-derived products approved in March 2026 also went through PMDA review before receiving conditional and time-limited approval. This profile looks at one question only: how much influence the PMDA actually has over iPS cells and regenerative medicine.
What this article covers
- The PMDA in 30 seconds
- What kind of body it is: the four types of influence
- What it does for iPS cells and regenerative medicine
- How far its influence reaches (powers and geography)
- Companies and organisations it works with
- Outlook
- Glossary, references and claim-to-source audit
1. The PMDA in 30 seconds
FULL NAMEPharmaceuticals and Medical Devices AgencyAn incorporated administrative agency. Usually abbreviated PMDA
COUNTRYJapanLocated in Kasumigaseki, Chiyoda-ku, Tokyo
TYPE OF BODYIncorporated administrative agencyManaged against medium-term objectives. The supervising minister is the Minister of Health, Labour and Welfare. Not a government ministry
ESTABLISHED1 April 2004Formed by merging the work of three earlier bodies, including the former drug review centre
STAFF1,053As of 1 April 2025. The published documents give no figure for staff dedicated to regenerative medicine products
REVIEW PERFORMANCE8.9 months (priority products)Total review time for regenerative medicine products (FY2025, median). Standard products: 11.1 months
ROLE IN iPS CELLS AND REGENERATIVE MEDICINEConsultation, review and site inspectionThe Minister of Health, Labour and Welfare decides approval. The PMDA is the reviewing side
REACHEvery product sold in JapanOverseas manufacturing sites can be inspected too. It also takes part in international meetings
The one thing to grasp first
The PMDA is the working agency that actually reviews regenerative medicine products, including iPS cell-derived ones, and gives developers advice, but the Minister of Health, Labour and Welfare decides whether to approve. The PMDA's own guide says that approval of a regenerative medicine product is granted by the Minister and that applications are submitted at the PMDA's counter Sourced. The two iPS cell-derived products approved on 6 March 2026, Cuorips' ReHeart and Sumitomo Pharma's AMCHEPRY, were approved inside this system as conditional and time-limited approvals Sourced. (In Japan, a "regenerative medicine product" is a legal category under the Pharmaceuticals and Medical Devices Act that covers processed cell products and gene therapy products.)
2. What kind of body it is: the four types of influence
Unlike the companies in this series, the institutions that appear in coverage of iPS cells and regenerative medicine do not make cells themselves. What separates them is the route by which they influence the industry, and that gives four types.
3. What it does for iPS cells and regenerative medicine
For regenerative medicine products (cells that have been processed, for example by culture, and gene therapy products), the PMDA carries out the following work Sourced.
| Role | What it involves | Document behind it |
|---|---|---|
| Development consultations | Even before a clinical trial, it offers face-to-face consultations, pharmaceutical regulatory strategy consultations and a 30-day investigation of the initial clinical trial plan notification for cell and tissue-processed products. In FY2025 it held visiting consultations with 56 Japanese and 23 overseas companies | The PMDA's page on regenerative medicine products, and the business performance report |
| Approval review | It reviews a product's efficacy and safety. Total review time for regenerative medicine products approved in FY2025 (median) was 8.9 months for priority products and 11.1 months for standard products | The business performance report |
| Conditional and time-limited approval | A system under which a regenerative medicine product that is not homogeneous can be approved early, with conditions and a time limit, once its efficacy is presumed. The PMDA handles pre-application consultation and review | The guidance on conditional and time-limited approval, and the handling notification |
| SAKIGAKE designation | A system that gives priority in consultation and review to regenerative medicine products that meet conditions such as being groundbreaking. The Minister makes the designation, and the PMDA works on it together with the MHLW | The PMDA's page on the SAKIGAKE designation system |
| Inspection of manufacturing sites | It carries out on-site inspections for GCTP (the standard for manufacturing and quality control of regenerative medicine products). In FY2025 there were eight (three overseas, five in Japan) | The business performance report |
| Inspection for licences on the clinical side | Under the Act on the Safety of Regenerative Medicine, it inspects the structure and equipment of facilities that manufacture specified processed cells, and carries out on-site visits. The scope widened after the law was amended in May 2025 | The PMDA's page on conformity inspections, and the business performance report |
| International harmonisation | It takes part in ICH Q5E, the discussion group on cell and gene therapy, and a working group of the IPRP (the International Pharmaceutical Regulators Programme). It also supports regulatory development in Asia and Africa | The business performance report |
A materials engineer's view: a system built on the premise that products are "not homogeneous"
One requirement for conditional and time-limited approval is that the regenerative medicine product in the application is not homogeneous Sourced. A product made from living cells will have properties that vary with the lot and the raw materials, and that is hard to avoid, so this premise reads as a reflection of that (our interpretation). An MHLW notification, drawing on experience from PMDA consultations and reviews, advises applicants to discuss their evaluation plan before applying Sourced. For iPS cell-derived products, two things become the focus of review: control of the manufacturing process, and the plan for confirming efficacy after approval.
The list of products approved on 6 March 2026 includes the following two, classified as human induced pluripotent stem cell-processed products Sourced.
| Brand name (company) | What it is (as the PMDA describes it) | Form of approval |
|---|---|---|
| ReHeart (Cuorips) | Human (allogeneic) iPS cell-derived cardiomyocyte sheets. For severe heart failure caused by ischaemic cardiomyopathy that does not respond adequately to standard treatment | Conditional and time-limited approval; designated an orphan regenerative medicine product |
| AMCHEPRY (Sumitomo Pharma) | Raguneprocel. A mass of dopaminergic neural progenitor cells made from iPS cells, transplanted into the brain. For Parkinson's disease that does not respond adequately to existing drug therapy | Conditional and time-limited approval; orphan designation; SAKIGAKE designation |
4. How far its influence reaches (powers and geography)
| Aspect | What the PMDA has | What it does not have |
|---|---|---|
| Powers | Consultation, review and inspection of manufacturing sites for regenerative medicine products. Its review results form the basis on which the Minister decides approval | The final decision on approval, SAKIGAKE designation and accreditation of foreign manufacturers (all made by the Minister of Health, Labour and Welfare). Allocation of research funding |
| Geography | Products sold in Japan. Overseas manufacturing sites can be inspected, and a foreign manufacturer needs the Minister's accreditation to sell in Japan. It takes part in international meetings such as ICH and supports regulatory development in Asia and Africa | Decisions on approvals in other countries. Authority to change other countries' systems |
| Scale | 1,053 staff (1 April 2025). Review times of 8.9 months for priority products and 11.1 months for standard products (FY2025) | Staff numbers or budget for regenerative medicine products alone (no separate figure in the published documents) |
| International reference point | The report says the PMDA worked on "creating the conditions for introducing the Japanese regulatory model" (through bilateral meetings and similar) | Whether other countries adopt that model |
A note on reading the numbers
The staff figure covers the PMDA as a whole, including drugs, medical devices and relief for adverse drug reactions Sourced. The review times are the total period from application to approval, and in some cases they exclude the time applicants spend preparing documents. Also, conditional and time-limited approval has a different standing from ordinary approval: efficacy has to be confirmed within the time limit, and a fresh application made Sourced.
5. Companies and organisations it works with
In Japan's approval of regenerative medicines, deciding, reviewing and funding research are done by different bodies. This section shows the division of work that can be confirmed from PMDA documents.
Division of roles
| Organisation | Role for regenerative medicine products | Source |
|---|---|---|
| Minister of Health, Labour and Welfare (MHLW) | Decides on marketing approval, SAKIGAKE designation and accreditation of foreign manufacturers. For conditional and time-limited approval, seeks the council's opinion. The MHLW also issues guidance and notifications | References 6, 7, 8, 9 and 11 |
| PMDA | Consultation, approval review, inspection of manufacturing sites and safety measures. It also inspects the structure and equipment of overseas manufacturing sites | References 1, 3, 8 and 11 |
| Pharmaceutical Affairs and Food Sanitation Council | Gives the Minister its opinion on conditional and time-limited approvals | Reference 6 |
| AMED | Runs research grant programmes. The MHLW's regenerative medicine page points to AMED's programme page | Reference 13 |
| Applicant companies | Cuorips, Sumitomo Pharma and others appear as applicants in the lists of approved products | Reference 4 |
6. Outlook
| What the evidence suggests | Primary information it rests on | Category |
|---|---|---|
| Conditional and time-limited approval has had its procedure set out in writing, in the March 2024 guidance and the October 2025 notification, so it is likely to be used more readily in future applications for regenerative medicine products | The guidance and the handling notification explain pre-application consultation, drawing up an evaluation plan, and the application categories | Inference |
| Whether the two iPS cell-derived products given conditional and time-limited approval in March 2026 win full approval within their time limits | The law says the time limit is set at no more than seven years. This article has not verified each product's time limit or the content of its approval conditions | Not yet confirmed |
| When the next iPS cell-derived product will be approved | The PMDA's published documents give no outlook on reviews under way | Not yet confirmed |
7. Glossary
- Regenerative medicine product
- A category under the Pharmaceuticals and Medical Devices Act: cells that have been processed, for example by culture, for use in rebuilding the body's structure or function, together with gene therapy products.
- Conditional and time-limited approval
- A system under which a regenerative medicine product that is not homogeneous can be approved early, with conditions and a time limit of no more than seven years, once its efficacy is presumed.
- SAKIGAKE designation
- A designation by the Minister for regenerative medicine products that meet conditions such as being groundbreaking, which then receive priority in consultation and review.
- GCTP
- Good Gene, Cellular, and Tissue-based Products Manufacturing Practice, the standard for manufacturing and quality control of regenerative medicine products. The PMDA inspects manufacturing sites against it.
- Pharmaceutical Affairs and Food Sanitation Council
- An advisory council that gives opinions to the Minister of Health, Labour and Welfare. Its opinion is sought for conditional and time-limited approvals.
8. References
- PMDA About the PMDA (in Japanese): name, establishment on 1 April 2004, status as an agency managed against medium-term objectives, and its three areas of work. https://www.pmda.go.jp/about-pmda/outline/0001.html
- PMDA Business performance report for FY2025 (in Japanese): 1,053 staff (1 April 2025), supervising minister, location, review times, GCTP on-site inspections, international harmonisation and visiting consultations. https://www.pmda.go.jp/files/000282524.pdf
- PMDA Page on approval review of regenerative medicine products (in Japanese): definition, list of notifications and guidance, and the 30-day investigation. https://www.pmda.go.jp/review-services/drug-reviews/about-reviews/ctp/0007.html
- PMDA List of approved products for FY2025, regenerative medicine products (in Japanese). https://www.pmda.go.jp/files/000280052.pdf
- PMDA List of approved products for FY2026, regenerative medicine products (in Japanese). https://www.pmda.go.jp/files/000282058.pdf
- MHLW (published on the PMDA site) Guidance on conditional and time-limited approval of regenerative medicine products and on drawing up a subsequent efficacy evaluation plan (in Japanese, 29 March 2024): the text of Article 23-26 of the Pharmaceuticals and Medical Devices Act and the time limit of no more than seven years. https://www.pmda.go.jp/files/000267914.pdf
- MHLW (published on the PMDA site) Notification on the handling of conditional and time-limited approval of regenerative medicine products (in Japanese, 6 October 2025, Iyakukishinhatsu 1006 No. 1). https://www.pmda.go.jp/files/000277559.pdf
- PMDA Procedure for marketing regenerative medicine products (in Japanese): approval by the Minister of Health, Labour and Welfare, with applications submitted at the PMDA's counter. https://www.pmda.go.jp/files/000216666.pdf
- PMDA The SAKIGAKE designation system for drugs, medical devices, in vitro diagnostics and regenerative medicine products (in Japanese). https://www.pmda.go.jp/review-services/drug-reviews/0002.html
- PMDA Conformity inspections under the Act on the Safety of Regenerative Medicine (in Japanese): inspections for manufacturing licences for specified processed cells and similar products. https://www.pmda.go.jp/review-services/gmp-qms-gctp/ensure-safety-regen/0001.html
- PMDA Applying for accreditation as a foreign manufacturer of regenerative medicine products (in Japanese): accreditation by the Minister, with inspection of structure and equipment by the PMDA. https://www.pmda.go.jp/review-services/drug-reviews/foreign-mfr/0011.html
- PMDA List of approved new drugs for FY2025 (in Japanese): the explanation that the Minister of Health, Labour and Welfare approves after PMDA review and consultation with the council. https://www.pmda.go.jp/review-services/drug-reviews/review-information/p-drugs/0039.html
- MHLW Regenerative medicine, cell medicine and gene therapy (in Japanese): the link to AMED's regenerative medicine programme page, and the Act on the Safety of Regenerative Medicine. https://www.mhlw.go.jp/stf/seisakunitsuite/bunya/kenkou_iryou/iryou/saisei_iryou/index.html
9. Claim-to-source audit
| Claim in the article | Category | Source |
|---|---|---|
| Full name, establishment on 1 April 2004, and status as an agency managed against medium-term objectives | Sourced | Reference 1 https://www.pmda.go.jp/about-pmda/outline/0001.html |
| 1,053 staff (1 April 2025), the Minister of Health, Labour and Welfare as supervising minister, and the location | Sourced | Reference 2 https://www.pmda.go.jp/files/000282524.pdf |
| Total review times for regenerative medicine products (8.9 months for priority products, 11.1 months for standard products), eight GCTP on-site inspections, visiting consultations and international harmonisation work | Sourced | Reference 2 https://www.pmda.go.jp/files/000282524.pdf |
| The definition of regenerative medicine products, the notifications and guidance, and the investigation of the initial clinical trial plan notification | Sourced | Reference 3 https://www.pmda.go.jp/review-services/drug-reviews/about-reviews/ctp/0007.html |
| The content and form of approval of the FY2025 approved products (including ReHeart and AMCHEPRY) | Sourced | Reference 4 https://www.pmda.go.jp/files/000280052.pdf |
| Products approved from April 2026 onwards | Sourced | Reference 5 https://www.pmda.go.jp/files/000282058.pdf |
| The requirements for conditional and time-limited approval, and the time limit of no more than seven years (Article 23-26 of the Act) | Sourced | Reference 6 https://www.pmda.go.jp/files/000267914.pdf |
| Pre-application consultation for conditional and time-limited approval, and the application categories | Sourced | Reference 7 https://www.pmda.go.jp/files/000277559.pdf |
| Approval of regenerative medicine products by the Minister of Health, Labour and Welfare, with applications submitted at the PMDA's counter | Sourced | Reference 8 https://www.pmda.go.jp/files/000216666.pdf |
| The pointer to AMED's research grant programme, and the amendment of 31 May 2025 to the Act on the Safety of Regenerative Medicine | Sourced | Reference 13 https://www.mhlw.go.jp/stf/seisakunitsuite/bunya/kenkou_iryou/iryou/saisei_iryou/index.html |
| SAKIGAKE designation being made by the Minister, with the PMDA working on it together with the MHLW | Sourced | Reference 9 https://www.pmda.go.jp/review-services/drug-reviews/0002.html |
| Inspection of the structure and equipment of facilities making specified processed cells, and on-site visits | Sourced | Reference 10 https://www.pmda.go.jp/review-services/gmp-qms-gctp/ensure-safety-regen/0001.html |
| Accreditation of foreign manufacturers by the Minister, with the PMDA inspecting structure and equipment | Sourced | Reference 11 https://www.pmda.go.jp/review-services/drug-reviews/foreign-mfr/0011.html |
| The route in which the Minister approves after PMDA review and the council's deliberation (as explained for new drugs) | Sourced | Reference 12 https://www.pmda.go.jp/review-services/drug-reviews/review-information/p-drugs/0039.html |
| The four types of institution, and the diagram of the approval route | Our calculation | Our own classification and diagram (drawn from references 6, 8 and 12) |
| The "not homogeneous" requirement assuming variability in the quality of cell products | Inference | This article's reading of the wording of the requirement in reference 6 |
| Conditional and time-limited approval becoming easier to use in future applications | Inference | This article's reading of the groundwork laid in references 6 and 7 |
| Whether the two products approved in March 2026 win full approval within their time limits | Not yet confirmed | This article has not verified each product's time limit or approval conditions |
| When the next iPS cell-derived product will be approved, and the staff numbers or budget for regenerative medicine products alone | Not yet confirmed | Not stated in references 1 to 5; this article does not estimate them |
Last updated 1 October 2026 / Troy Technical
All figures come from the PMDA's official website and business performance report and from MHLW notifications. Items marked "Inference" are this article's reading of the primary information. This article is not medical advice.