What Is and Is Not FDA Approved in Regenerative Medicine

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Sources on this page: www.fda.gov · www.accessdata.fda.gov

What Is and Is Not FDA Approved in Regenerative Medicine

There are four phrases in wide use, and three of them are not approval.

Approved products in this field do exist, and the list is narrower than the marketing around it. FDA puts the second half of that plainly:

“There continues to be broad marketing of unapproved products considered regenerative medicine therapies that are intended for the treatment or cure of a wide range of diseases or medical conditions. These products require FDA licensure/approval to be marketed to consumers.”

Source: FDA, “Important Patient and Consumer Information About Regenerative Medicine Therapies.” Accessed 18 September 2026.

That is FDA’s sentence and we are not going to improve on it with a figure of our own, because we have not measured how much of the market sits where. Knowing which of the four phrases you are looking at is the difference between checking something and assuming it.

Four phrases that are not the same thing

A note on what follows. The four-way split below is our framing, not an FDA classification. FDA does not publish “regulated / registered / approved / cleared” as four official categories. We group them this way because these are the four phrases people actually hear, and separating them is what stops one from being mistaken for another. The facts inside each are FDA’s; the grouping is ours.

FDA regulated. This describes the agency’s authority over a category of products. It says nothing about whether any particular product has been reviewed or cleared. The phrase can be true and still carry very little information.

FDA registered facility. Establishments that handle certain products are required to register with the FDA and list what they handle. Registration is an administrative step. It is not a review of the product, and it is not an endorsement of anything the facility does.

FDA approved product. This is the one that means a specific product, for a specific use, went through review. It attaches to the product, not the clinic.

Where the products on the list got their approval, and why not everything in this field travels that road. For the products that appear on FDA’s licensed cellular and gene therapy list, the mechanism is a biologics license, granted on a Biologics License Application. FDA uses both words together for it, describing a complete application as one that satisfies “the high FDA standard for approval and licensure.” So if you see licensed where you expected approved, that is not a downgrade or a different tier; for a biological product it is the same event.

But this is not the only route in this field, and assuming it is will mislead you. Some cell and tissue products are regulated under a separate framework and do not go through a licence application at all; whether a given preparation does is exactly the question our page on the four criteria exists to walk you through. Others are regulated as devices and carry a clearance rather than an approval, which is the fourth phrase above. So the honest form of the question is not does it have a licence but which route is this on, and what document does that route produce?

Source: FDA, Biologics License Applications (BLAs) for CBER-Regulated Products. Accessed 18 September 2026.

FDA cleared. A fourth phrase, and it belongs beside the other three because it is easy to hear as a fourth grade of approval when it is not one. Clearance is a device decision. It runs through a different route from approval, and what it decides is different: whether a device is substantially equivalent to a device already on the market.

That matters in a specific way for this subject. Several preparations in this field are produced at the point of care by a machine (a centrifuge and a kit), and it is the machine that carries the clearance.

One record, read as it is written. We opened the 2002 clearance letter for a platelet separator, number K021902. FDA’s letter records the regulation as 21 CFR 862.2050, and names it:

“Regulation Name: General purpose laboratory equipment labeled or promoted for a specific medical use”

The classification name on the file is General Purpose Centrifuge for Clinical Use, the medical specialty is recorded as clinical chemistry, and the decision reads “Substantially Equivalent.”

Now the part that decides what the clearance is about. The Indications for Use on the same file describe a device that prepares plasma and platelet concentrate at the point of care, and then say what the output is for:

“The plasma and concentrated platelets produced can be used for diagnostic tests.”

Source: FDA 510(k) file K021902, clearance letter and 510(k) summary, decision date 12 August 2002. Accessed 18 September 2026.

Read that sentence rather than around it. The cleared indication speaks about diagnostic tests. It is not a statement that injecting the preparation treats any condition, and it names no condition at all.

A separate trap in the same record, and it is easy to fall into. That 2002 letter records the device as Regulatory Class II, under product codes FMF and JQC. If you look up product code JQC today, FDA’s classification database shows the category as Class I and 510(k) exempt. Those are two different states of the same code, twenty-odd years apart, and the proof that they cannot be the same is in front of you: a category that is exempt from 510(k) now could not have been the basis of a 510(k) clearance then. So do not read a code’s present-day classification back onto an old clearance. Read the clearance letter for what governed that decision, and the classification page for what governs the category now.

So “FDA cleared” can be entirely true and still be a statement about a piece of laboratory equipment, decided decades ago, under rules that have since changed. The question that separates them is short: what exactly was cleared, under which number, and what do its indications for use say? All three are free to look up.

The FDA has addressed the confusion between these phrases directly:

“patients and consumers are being referred to clinicaltrials.gov, or are told that a product is registered with FDA, as a way to suggest that the products being offered are in compliance with FDA laws and regulations. This is often false.”

Source: FDA, “Important Patient and Consumer Information About Regenerative Medicine Therapies.” Accessed 18 September 2026.

When you hear one of these phrases, the useful next move is to ask which of the four is meant, and then to ask it about the product rather than the building.

What an approved product looks like

Two examples, from different cell classes, both on the FDA’s current list of licensed cellular and gene therapy products.

MACI, listed as Autologous Cultured Chondrocytes on a Porcine Collagen Membrane. Autologous: the cells come from the patient. Cultured: the cells were grown outside the body before being returned. Chondrocytes: cartilage cells.

Here is its licensed indication, word for word:

“MACI is an autologous cellularized scaffold product indicated for the repair of symptomatic, single or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults.”

Source: FDA, “Approved Cellular and Gene Therapy Products.” Accessed 18 September 2026.

Read what that sentence excludes. Not cartilage generally: full-thickness defects. Not any joint: the knee. Not any patient: adults. An approval this specific is not a licence for knee pain.

RYONCIL, listed as remestemcel-L-rknd, approved 18 December 2024. This is a mesenchymal stromal cell product, and mesenchymal stromal cells are one of the cell classes named in FDA’s own enumeration of materials being marketed in this field.

Source for the date: FDA press announcement, “FDA Approves First Mesenchymal Stromal Cell Therapy to Treat Steroid-refractory Acute Graft-versus-host Disease,” for immediate release 18 December 2024; page stamp Content current as of: 12/18/2024. Accessed 18 September 2026. Note for anyone checking: the approval letter on the product page carries 19 December, one day later. The approval date is the 18th and the letter date is the 19th; if you see both, that is why.

Its licensed indication, word for word:

“Indicated for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older.”

Same source, accessed 18 September 2026.

Graft versus host disease is a complication of a stem cell transplant. Steroid-refractory means it did not respond to steroids. Pediatric patients aged two months and older.

That is the whole of it. And it points at a specific way this goes wrong: if someone tells you that mesenchymal stromal cells are FDA approved, that statement is about a class, and approval does not attach to a class. One MSC product is approved, for the indication quoted above. FDA’s own announcement of it uses the word first, which is a statement about one product entering the list rather than about the material becoming approved. Ask which product, and read what that product is licensed for.

These two examples matter for a reason that has nothing to do with whether either is right for anyone. Together they show two things at once. The approval pathway in this field is open and has been used, including for a product whose cells were cultured. And an approval, when it exists, is narrow enough to fit in a sentence you can read in ten seconds. When you read that a cell product would require premarket review, that is a description of a route, not a closed door.

Two FDA pages, two different answers

This next part is the most useful thing on this page, and we found it by doing exactly what we are asking you to do.

The FDA’s consumer alert on this subject says:

“Currently, the only stem cell products that are FDA-approved for use in the United States consist of blood-forming stem cells (also known as hematopoietic progenitor cells) that are derived from umbilical cord blood.”

Source: FDA, “Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes.” Accessed 18 September 2026.

The FDA’s own list of licensed products includes RYONCIL, a mesenchymal stromal cell product, approved on 18 December 2024. Mesenchymal stromal cells are not blood-forming stem cells from cord blood.

Both pages are live on fda.gov today, and as a current inventory of what is approved they are not aligned. We are putting it no more strongly than that, and the reason is worth knowing: FDA’s approval of RYONCIL calls it a mesenchymal stromal cell therapy, while the older consumer page is talking about stem cell products. The vocabulary in the two sentences is not identical, so the older sentence is not simply a mistake to be corrected. What it is, for your purposes, is not a complete list of what is approved today.

We are not resolving this for you and we are not suggesting anyone has done anything wrong. An agency’s pages are not all revised together, and consumer material is updated on its own schedule. What matters is the lesson sitting inside it:

A page being live does not mean it is current. A sentence written before a decision stays on the site after the decision. If you are checking an approval question, the list of licensed products is the record of what has been approved; a consumer explainer describes the situation as of whenever it was last revised.

So when you check, check the list, and look for a date on anything else.

Where the approved list lives, and how to read it

The FDA maintains a public page of approved cellular and gene therapy products. It is the place to check, and it changes, which is why no count appears on this page. A number written here today would quietly become wrong. Open the list and read it at the moment you need it.

When you get there, two columns matter together: the product name and what it is licensed for. A product name alone does not tell you what the license covers.

Products with no approved version

Some categories currently have no approved product at all. The FDA states one of them plainly:

“Exosome products are also regulated by FDA. As a general matter, exosome products intended to treat diseases or conditions in humans require FDA approval. There are currently no FDA-approved exosome products.”

Source: FDA, “Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes.” Accessed 18 September 2026.

If a product is described to you using the word exosome, or a trade name for an exosome preparation, that sentence is the relevant one to bring to the conversation.

Why approval is indication specific

This is where a check can fail even when someone actually performs it.

An approval is not granted for a disease name in general. It is granted for a defined use, and the two indications quoted above show how narrow that can be.

Put them side by side. Both products are approved. One is for full-thickness cartilage defects of the knee in adults. The other is for steroid-refractory acute graft versus host disease in children from two months old. Neither approval says anything about the other’s territory. And neither says anything about any use outside the indication written into it, whoever is offering that use and however it is described.

That is what indication specific means in practice.

Two products can both be described as approved and mean very different things for your situation, because the scope lives in the sentence after the product name. Finding a product on the list is the first half of the check. The second half is reading what the license actually covers and comparing it with what is being offered to you.

A product can be genuinely approved and still be outside its approved indication in the use you are being offered. That situation has a name, and FDA uses it:

“using an FDA-approved drug for an unapproved use (sometimes called an ‘off-label’ use)”

Source: FDA, Understanding Unapproved Use of Approved Drugs “Off Label”. Accessed 18 September 2026.

So there are two distinct situations and the word for the second one is off-label. Knowing the term is useful precisely because you will hear it, and because it is a description rather than a verdict: FDA’s own page discusses it as something that happens, not as something that is by definition wrong.

Which situation you are in is a fact about the proposal in front of you, and it is answerable. Ask whether the proposed use is within the labeled indication, and ask for the indication itself so you can read it. We are not telling you what follows from either answer; that is a clinical conversation. We are telling you that the two answers are different and that the question has an answer.

What to do with what you find

Your check ends in one of three places.

Approved for the stated indication. The product is on the list and the licensed use matches what is being proposed.

Approved, but for a different indication. The product is on the list and the licensed use is something else. This is a real and specific finding, and it is worth asking about directly.

Not found under the name you searched. Say it that way rather than “not approved”, because the two are not the same and the difference is checkable. A name can be a trade name, a generic name, a spelling variant, or a listing name different from the one you were given, and any of those returns nothing while an approved product exists. So before concluding anything, ask for the full product name in writing and search that.

If it is genuinely absent from the list, that is still not a verdict on the treatment. It means the approval question has a different answer, and the next question is which regulatory framework applies at all, asked before any particular one is opened. For a preparation that is an HCT/P, the four-criteria page is where that continues. A blood-derived preparation, a secreted body fluid, an exosome product and a preparation whose FDA record attaches to a device each follow a different branch, and our glossary sets out which materials sit outside the HCT/P framework and quotes the regulation that puts them there. Establish the branch before applying the questions that belong to one, because the questions do not transfer between branches.

Notice what none of these three say. None of them say whether the treatment is right for you. That is a clinical judgment, it depends on your history and your circumstances, and this page cannot make it. What the check gives you is the regulatory position, which is one input among several.

Where to go from here

If your product is not on the approved list, the next question is which category it sits in: The Four Criteria and the Exceptions That Decide How an HCT/P Is Regulated. If it is on the list but the indication does not match, the next question is what evidence exists for the use being proposed: Reading the Study a Clinic Shows You.

Before you leave this page: ask for the full product name in writing, then search the list yourself. If a written product name is not available, that is itself a piece of information worth recording.

What this does not mean

Being on the approved list does not mean a product will work for you, and being absent from it does not mean a product is useless or that offering it is unlawful. This page describes where to look and how to read what you find. It does not evaluate any treatment, any clinic, or any individual case.

Eight FDA records, and two of them disagree with each other. Every quotation above comes from one of the eight below, including the two whose conflict this page is built around. That is the strongest possible reason to open them rather than take our word: you can see the disagreement for yourself, dated, on the agency’s own site.

Written and fact-checked against the sources above on 18 September 2026. No independent expert review has taken place yet, and this page says so rather than implying one. This page is reviewed every three months and whenever the FDA approved products list changes.

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