Commercial-stage Cell Therapy Companies and Products

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Updated 2012-03-13 update

There are a number of products in the market which are made up entirely of cells or purport to have live cells in them and as such are included in our definition of cell therapy.

Some of these have received regulatory market approval.  However, a number of these products are being legally sold, even in highly regulated markets, without overt regulatory approval because they arguably meet the requirements set out for products which do not require such approval to be marketed.

Below is sample list of companies with cell therapy products* on the market in Europe, USA, or Japan.  In some instances there are reservations about whether or not these products still contain viable cells.  Our listing of these product is not an endorsement of their market claims nor of their strict regulatory compliance.

Company                                                    Product              
Advanced BioHealing (now part of Shire)    Dermagraft
AlloSource                                                 AlloStem
Altrika                                                        MySkin
Altrika                                                        CryoSkin
Alphatec Spine                                           PureGen
Amedica                                                     BioDfactor
Arthro Kinetics                                           CaReS
Avita Medical                                             ReCell® Spray-On Skin
Bio-Tissue                                                  Prokera
Bio-Tissue                                                  AmioGraft
BioTissue Technologies                               BioSeed-C
BioTissue Technologies                               chondrotissue
Cytori                                                         Celution System
Dendreon                                                   Provenge
euroderm                                                    Epidex
euroderm                                                    EpiGraft
Fidia Farmaceuitici                                      Hyalograft 3D
Fidia Farmaceuitici                                      Laserskin
Fidia Farmaceuitici                                      Hyalograft C
Fibrocell                                                      laViv
Genzyme (a Sanofi company)                      Epicel
Genzyme (a Sanofi company)                      Carticel
Genzyme (a Sanofi company)                      MACI Implant
Japan Tissue Engineering Co.                      J-TEC Epidermis
Japan Tissue Engineering Co.                      J-TEC Cartilage                                        
Japan Tissue Engineering Co.                      J-TEC Corneal Epithelium                        
Living Cell Technologies                              DIABECELL
NuTech                                                       NuCel
NuVasive                                                    Osteocel Plus
Organogenesis                                             Appligraf
Organogenesis                                             GINTUIT
Orthofix                                                      Trinity Evolution
TiGenix                                                       ChrondroCelect
Therakos                                                     Therakos Photopheresis

Most of these are only available in one or a few jurisdictions.  Sanofi's Genzyme products (Epicel & Carticel) are two of the few which are multi-nationally available.

South Korea is an interesting example of a country with a mature regulatory framework with more than its share of approval.  There have been 14 cell therapy products approved for sale in South Korea.  They are as follows:

Company                                                    Product    
Anterogen                                                  Adipocel
Anterogen                                                  Cupistem
Antrogen                                                    Queencell
Cellontech                                                  RMS ossron
Cellontech                                                  Chondron
Chabiotech                                                 Hyalgraft-3D+
Chabiotech                                                 Autostem
Creagene                                                    Creavax-RCC+
FCB-Pharmicell                                          Heartcellgram-AMI
Innocell                                                       Immuncell-LC+
Innomedisys                                                Innolak+
MCTT                                                        Keraheal+
Medipost                                                     Cartistem
NKBio                                                        NKM+
S-Bio Medics                                              Cureskin
Tegoscience                                                 Holoderm
Tegoscience                                                 Kaloderm

+ These products were brought to market in 2006-7 under a conditional approval unique to South Korea's regulation that allows commercial sale in certain instances while a pivotal trial is underway.

At present, the Korea-FDA is assessing a Biologics License Application (BLA) for CARTISTEM® being developed by Medipost. Once approved, CARTISTEM® will become available as the World's first hUCB-MSC derived adult stem cell drug for treating osteoarthritis and will be marketed in Korea in association with Dong-A pharmaceuticals

* This list does not purport to be exhaustive of all cell therapy products legally sold in these regions.  This list  does not include approved products in other highly-regulated jurisdictions, such as Australia, New Zealand, or Singapore, for example.  This list also excludes those cell-based treatments provided as a hospital or clinic-based service such as stem cell transplantation (hospital) or Regenexx (Regeneration Sciences, Inc.).

For the purposes of this list, “cell therapy” is defined loosely as any product which has in it live cells when administered to the patient including tissue transplants and devices.

Note that some of these products may be subject to emerging regulatory restrictions under the EMA ATMP regulations which may result in them having to be pulled from the market by the end 2012 at the latest.

Several of the products on this list did not receive overt regulatory approval but are being sold in regulated markets under exemption provisions which allow companies to bring cell therapy products to market which meet certain strict criterion.  At least a couple of the products listed are the subject of dispute between the company and the FDA or other regulatory agency as to whether or not they qualify under the 'exemptions'.

If you would like to suggest any revisions or additions to this list, please do so in the comment section below.

Commercially Available Cell Therapies?

The week of December 2, 2008 I posed the following one-question survey:

Survey
1. How many unique cell therapy* products** do you believe are currently commercially available*** in the world?

* "cell therapy" is defined as any cell-based product involving live cells 'produced' ex vivo and used as part of the therapeutic (not just to create the product) including cell-mediated gene therapies.

** "products" is meant to exclude "mere" stem cell transplantation for any and all indications thus excluding the uses of SCT in regulated markets as well as unregulated SCT services - a "unique product" is one which is distinguishable in a material, technical way from another product (e.g., not just the same product marketed under a different name in different countries).

*** "commercially available" is meant to include any "product" a consumer can purchase whether or not it is part of a clinical trial (given that in some countries these are not mutually exclusive).

I distributed the survey to members of the following groups:

• those who follow me on Twitter (currently a group 66 individuals sufficiently interested in cell therapy to follow my postings);
• my connections on LinkedIn (a group of 203 professionals in or interested in the cell therapy sector);
  
• the 110+ or so members of the LinkedIn Cell Therapy Industry Group;
• readers of this Blog; and
  
• the roughly 55 members of ISCT's cell therapy commercialization committee.

There was no control on multiple responses and no restriction on participation. Participants were forced to choose only one answer among responses ranging from 1-10 to 71-80 in increments of 10 (e.g., 1-10, 11-20, etc).

Data, Assumptions, & Observations
There have been 8 responses to-date. This is a small n but I can't justify spamming my audience any more than I have so we'll have to work with this for now.

If we assume that only members of the groups listed above participated in the survey, then it is reasonable to characterize the participants, at the very least, as informed participants who know more about the cell therapy industry than the average person. I will also assume that participants only voted once.

25% (2 responders) believe there are 10 or fewer cell therapy products commercially available per my definitions. Another 25% responded 11-20. One person thought there were 30-something and another 40-something. A final 25% thought there were 50-some cell therapy products (as I defined them) currently commercially available worldwide.

Once again the quick and easy conclusion is that there is a significant lack of clarity around the current size or maturity of the cell therapy industry even amongst those in or following it closely and who consequently are - at the very least - a lot more informed about it than most.

Analysis
This is admittedly not an easy question to answer because so much hinges on the definition of "cell therapies", whether or not to include the various types of stem cell transplantation products and/or services, and what constitutes a "unique" product (e.g., whether one autologous chondrocyte transplant product for cartilage repair is truly distinguishable from another and how one would define such distinctions).

Let's look at my data. I am currently tracking 700+ companies which I define as stakeholders in the cell therapy sector. This includes ~250 therapeutic companies [i] with ~340 cell-based therapeutic products in the market or in some stage of clinical or pre-clinical development [ii].

Approximately 67 of these 'products' are commercially available in at least one country. There are a number of observations worth noting about these 'products'.

1. Not all of these 'products' have been 'approved' in 'regulated markets'. Some are simple stem cell transplants such as we see in the kind of unregulated clinics discussed in the recent issue of Cell Stem Cell (see the ISSCR Commentary article and a Correspondence article assessing the online portrayal of commercial stem cell transplantation retail ventures providing treatments in unregulated markets). Approximately 17 of the 67 are these unregulated stem cell transplantation “products” where companies are transplanting stem cells in clinics outside the US for a wide variety of diseases with little oversight or published data. I've assumed each such "clinic" has only one product (although that's almost impossible to discern in most instances) and I am certain there are more than 17 such clinics but accurately tracking them is not really one of my goals here.
   
2. Roughly 14 of the 67 are very similar skin (e.g., wound repair/regeneration) products available in various countries;
3. Approximately 10 of the 67 are very similar cartilage repair products available in various countries – many of which are autologous chondrocyte transplant (ACT) products; and
   
4. In some instances, there are multiple inclusions of the same product commercially available through different companies in different countries sometimes under different brand names.

By way of external reference, in his recent publication in Tissue Engineering, M. Lysaght cites 47 companies with at least one commercial product and Proteus Venture Partners often refers to “over 25 FDA-approved cell therapies”.

My analysis of the data leads me to conclude that there are currently ~50 cell therapy products commercially available worldwide. Approximately 26 of these are uniquely distinguishable product types. Roughly 20% (depending on your interpretations) are cell therapies available in the United States. These include extracorporeal photopheresis (ECP) therapy performed using the UVAR XTS, Fusionary, Osteocel\Trinity, Epicel, Carticel, OrCel, Prokera, AmnioGraft, Dermagraft, and others.

Summary
50% of the respondents to my admittedly-small survey believe there are less than 20 unique cell therapy products commercially available worldwide. None selected the 20-some answer (the correct answer according to my data and analysis). From the highly-informed group of respondents, answers ranged from 1-60 products with a full 50% of respondents split evenly between the extreme opposite ends of the spectrum (1-10 vs 51-60).

Conclusion
Regardless of whether my numbers are right, the lack of consensus among those in the sector regarding the size and maturity of the cell therapy industry - or at the very least how one defines it - is clear. For those of us in the industry...it's up to us to fix this. More on that in an upcoming post...

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[i] Estimates range from ~225 to ~300 therapeutic companies among what I define as ~700 stakeholder companies in the cell therapy industry. Sources: author’s database; Cell Therapy Pages (Connexon Communications); Proteus Venture Partners; Bionest Partners. Cell therapies and tissue engineering. February 2007; Burger SR. 2004. Cell and Gene Therapy - Challenges and Strategies for an Emerging Industry. Cell and Gene Therapy 5:9-14.

[ii] “Pre-clinical development” is defined to product in development prior to initiation of a phase I trial but not including products in the early research phase.

Cell Therapy Market Overview

Below is my snapshot of the cell therapy sector and year-to-date activity - investments, business activity, and market size - that I just posted on docstoc and on my website. It's a very basic status overview of the business side of the industry. There is much more detail behind these summary slides but I can't give away everything now can I? :)


Cell Therapy Business-Market Summary (July 2008)

-- Lee Buckler
the Cell Therapy Group