List of FDA Approved Cell and Gene Therapies

By Professor Paul Knoepfler, Ph.D. / 37 Comments / March 12, 2021 / FDA Approved Cell and Gene Therapies, FDA approved stem cell therapies / FDA, Informational

What are the current FDA Approved Cell and Gene Therapies?

In the past many of us have wished there was a list of FDA-approved stem cell therapies. Patients and fellow scientists often asked me, but I couldn’t find a list.

As a result, I made a comprehensive list of FDA Approved Cell and Gene Therapies, which is the focus of today’s post. I’ve updated the list now in 2024 with the great news that Casgevy and Lyfgenia, gene therapies for sickle cell, are now approved as of late 2024. It’s exciting that the FDA has approved about 40 gene and cell therapies.

What is in this article?

FDA and cell therapies

Other than what I mentioned above, there was nothing else approved that was an actual stem cell therapy. There were, however, cellular therapies approved even if not related to stem cells. In a way these fit into the broader category of regenerative medicine.

Note that you can watch me discuss this post in a new YouTube Video below.

List of diseases treated by stem cells & FDA approved

What about today in 2022? Before we get to the approved products, I made a short list of the diseases treated by stem cells.

Note that only very specific products matched with specific forms of these diseases have the FDA OK. For instance just because damaged cartilage is on the list doesn’t mean anything goes. There’s no approval to treat it with fat stem cells, bone marrow, umbilical cord cells or exosomes.

Melanoma
Mantle Cell Lymphoma
Large B-Cell Lymphoma
B-Cell ALL
Prostate Cancer
Some other kinds of cancer
Facial wrinkles (specific type)
Receding gums
Damaged cartilage
Leber congenital amaurosis (a retinal disorder)
SMA or spinal muscular atrophy

The list of diseases has been growing. What about the products?

FDA Approved Stem Cell Therapies, key context

We need to turn to the FDA itself for the clearest answer on where things stand now.

Too often stem cell clinics claim that what they offer is “FDA approved”. In actuality at best what some of them sell is not FDA approved, but rather technically compliant with the rules for 361 products. What this means in English is that the products are not regulated as drugs.

So what does the FDA have to say about what they have approved?

fda approved stem cell therapies — FDA has regularly warned about unproven stem cells

In mid-2020, they issued an advisory, “Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes.” In it they wrote:

“Stem cell products are regulated by FDA, and, generally, all stem cell products require FDA approval. 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. These products are approved for use in patients with disorders that affect the production of blood (i.e., the “hematopoietic” system) but they are not approved for other uses.”

This fits with the general boilerplate some of us in the stem cell field have used, as noted earlier.

Note that, of course, the US and the FDA are not the only ones in this arena. The Alliance for Regenerative Medicine has what we might call an international list of cleared cell and gene therapies here. They are a great organization.

Cell and gene therapies

The good news today is that the agency has an actual list that is publicly available. Even better news is that there are more approved cell therapies than last time I tried to tackle this topic.

An interesting side note is that the agency for several years now has been combining together cell and gene therapies into one category. This is also reflected in their Regenerative Medicine Advanced Therapy (RMAT) designation program, which contains both types. RMATs still have to go through the trial approval process but get special consideration. You can see a help infographic below that my intern Mina made of the clinical trial approval process.

The formal FDA list of approved drugs made from stem cells is called, appropriately enough, “Approved Cellular and Gene Therapy Products.”

The current list is up to date as of September 1, 2022. I’m going to update this post as the agency updates their list. It’s interesting to speculate on how different this list might be in as short as 5 years given the exciting clinical trials that are ongoing now. In 10 years I predict it’s going to be a much longer list.