This list, though not exhaustive, exclusively includes those products that have undergone the necessary scientifically-based, rigorous regulatory approval process, including in-human clinical trials where appropriate, overseen by internationally-recognized regulatory agencies, such as the U.S. FDA; the EU’s European Medicines Agency; Japan’s Pharmaceuticals and Medical Devices Agency; South Korea’s Ministry of Food and Drug Safety, among others.
If you have questions about or suggested edits/additions to this list, please contact Lyndsey Scull
Cell-Based Immunotherapy Products
Kymriah is a CAR T-cell therapy product indicated for the treatment of acute lymphoblastic leukemia, chronic lymphoid leukemia and diffuse large B-cell lymphoma in patients up to 25 years of age, as approved by U.S. FDA in August 2017, and the treatment of adult patients with relapse/refractory (r/r) large B-cell lymphoma, as approved by U.S. FDA in May 2018. It was also approved for these indications by the EC in August 2018, and by Health Canada in September 2018. It was approved in Japan for the treatment of ALL in February 2019.
Yescarta is a CAR T-cell therapy product indicated for the treatment of B cell malignancies such as non-Hodgkin lymphoma, acute lymphoblastic leukemia, mantle cell lymphoma, chronic lymphoid leukemia and diffuse large B-Cell lymphoma. Approved by U.S. FDA in October 2017. Approved by the EC in August 2018. Approved by Health Canada in February 2019.
An allogeneic therapy using T-cells genetically modified with a retroviral vector for the treatment of Graft vs. Host Disease, Hematopoietic Stem Cell Transplantation. Approved by EMA in August 2016.
Autologous Cellular Immunotherapy for the treatment of asymptomatic or minimally symptomatic metastatic castrate resistant (hormone refractory) prostate cancer. BLA approved by the FDA in 2010.
A medicine for anticancer cell immunotherapy, made with T-lymphocyte incubated and activated after extraction from the blood of a patient. Conditionally approved in S. Korea in June 2007.
Gene Therapy Products
LUXTURNA is an adeno-associated viral vector gene therapy indicated for the treatment of RPE65-mediated inherited retinal dystrophies. Approved by U.S. FDA in December 2017; pending MAA in Europe as of August 2017.
Strimvelis is an ex-vivo stem cell gene therapy which uses retroviral vector encoding adenosine deaminase gene transfer into hematopoietic stem/progenitor cells. Strimvelis is indicated for the treatment of adenosine deaminase severe combined immune deficiency. Approved by the EMA in May 2016.
IMLYGIC is a weakened form of Herpes Simplex Virus Type 1, which is commonly called the cold sore virus, indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery. Approved by EMA in December 2015, FDA in October 2015.
Shenzhen SiBiono GeneTech
A recombinant adenovirus engineered to express wildtype-p53 (rAd-p53), designed to treat patients with tumors which have mutated p53 genes. Gendicine is the first gene therapy product approved for clinical use in humans, approved in China in 2003.
A gene therapy consisting of an intramuscular injection of plasmid (DNA) coding for Hepatocyte Growth Factor (HGF), intended to grow blood vessels, for the treatment of critical limb ischemia. Approved in Japan in February 2019.
Cell Therapy Products
An allogeneic stem cell therapy to treat complex perianal fistulas in patients with Crohn’s disease. Approved by the EC in March 2018.
Stempeutics Research Pvt
An ex-vivo cultured adult allogeneic mesenchymal stromal cell therapy for the treatment of Critical Limb Ischemia. Conditionally approved in India in 2017.
JCR Pharmaceuticals Co Ltd, licensee of Mesoblast Ltd
TEMCELL is an allogeneic mesenchymal stem cell product indicated for the treatment of acute radiation injury, chronic obstructive pulmonary disease, Crohn’s disease, graft-versus-host disease, Type I diabetes and myocardial infarction. Fully approved by the Japanese Ministry of Health, Labour and Welfare in October 2015 and conditionally approved in Canada & New Zealand (also known at Prochymal).
Holoclar is a cell therapy based on autologous cultures of limbal stem cells. It regenerates a functional corneal epithelium allowing recovery of visual acuity. Holoclar is indicated for the treatment of moderate to severe limbal stem cell deficiency due to ocular burns. Granted conditional marketing authorization by the European Commission in February 2015.
An autologous bone marrow mesenchymal stem cell therapy approved in S. Korea in 2014 for the treatment of ALS.
An autologous adipose derived mesenchymal stem cell treatment to reduce inflammation and regenerate damage joint tissues, indicated for the treatment of Crohn’s fistula. Approved for marketing by South Korea’s Food and Drug Administration in July 2012.
A cellular therapeutic agent containing allogeneic human umbilical cord blood-derived mesenchymal stem cells, indicated for the treatment of knee cartilage defects such as traumatic articular cartilage, degenerative arthritis and rheumatoid arthritis. Approved for marketing by South Korea’s Ministry of Food and Drug Safety in January 2012.
An autologous intracoronary bone marrow-derived mesenchymal stem cell injection for the treatment acute myocardial infarction. Approved for marketing by South Korea’s Food and Drug Administration in July 2011.
Hemacord / ALLOCORD / HPC Cord Blood
SSM Cardinal Glennon Children’s Medical Center
The U.S. FDA’s only approved stem cell product to-date, a cord blood-derived product used for specified indications in patients with disorders affecting the body’s blood-forming system. For use in unrelated donor hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment. Approved in 2011, name change to HPC Cord Blood in 2013.
An allogeneic cell therapy for deep second degree burns and diabetic foot ulcers. Approved in S. Korea for burns in 2005 and diabetic foot ulcers in 2010.
An autologous mesenchymal stem cell for the treatment of connective tissue disorders. Approved in S. Korea in 2010.
A permanent skin replacement product grown from a patient’s own skin cells. The autologous keratinocytes are co-cultured with irradiated murine cells to form cultured epidermal autografts (CEA). Epicel is indicated for treatment of deep dermal or full thickness burns. Epicel has been used in the U.S. and other countries since 1988; approved in the United States in 2007 as a Humanitarian Use Device (HUD) under a Humanitarian Device Exemption (HDE).
An autologous bone cell implantation for the treatment of bone defects in patients caused by degeneration, drugs, intense physical stress, diet, genetics, obesity, smoking, alcohol or disease. Approved in S. Korea in 2009, approved in India in 2017.
Autologous Cultured Chondrocytes on a Porcine Collagen Membrane / Vericel
An autologous cellularized scaffold product, indicated for the repair of single or multiple symptomatic, full-thickness cartilage defects of the knee with or without bone involvement in adults. Approved by the U.S. FDA in December 2016.
An advanced bilayer dermal regeneration matrix for the treatment of diabetic foot ulcers. Approved by the FDA in 2016.
A cardiovascular scaffold which facilitates endogenous stem cells and other cells to regenerate and repair damaged tissue for the treatment of cardiovascular abnormalities. Marketed in the U.S. under 510(k) clearance as of 2014; marketed in Europe under CE Mark as of 2013; received medical device license in Canada in 2014; approved in Singapore in 2015.
Spherox (formerly Chrondosphere)
A product containing spheroids of human autologous chondrocytes for use in cartilage defects. Marketed in Europe as of 2017; marketed in Germany since 2007.
An autologous cell harvesting device that enables a to create a regenerative epithelial suspension using the sample of the patient’s skin for the treatment of skin discoloration. Marketed in Europe as of 2016.
A device composed of homogenous human recombinant type I collage for use in wound treatment. Marketed in Europe as of 2016.
A matrix made of collagen type I for use in the treatment of connective tissue disorders. Marketed in Europe as of 2016.
An autologous cell harvesting device that enables a to create a regenerative epithelial suspension using the sample of the patient’s skin for the treatment of ulcers. Marketed in Europe as of 2015.
A biodynamic hematogel composed of platelet-rich plasma gel prepared from a small sample of a patient’s own platelets and plasma as a catalyst for healing indicated for the treatment of wounds. Marketed in the U.S. under Section 361 for the Platelet Rich Plasma and under 510(k) clearance as of September 2007.
CHA Bio&Diostech Co Ltd
A cell therapy which cultivates autologous skin fibroblasts in 3D scaffolds formed of hyaluronic acid derivatives for the treatment of diabetic foot ulcers. Conditionally approved in S. Korea in July 2007.
A cultured epidermal autograft composed by culturing autologous keratinocytes. It is transplanted to the wound and aids in regeneration of the dermis and develops into new skin. Holoderm is indicated for the treatment of skin disorders such as burns, vitiligo, nevi and scars. Approved in S. Korea in 2002.
A tissue-engineered skin substitute made from a nylon mesh and a silastic semi permissible and biocompatible layer for the treatment of Epidermolysis Bullosa. Approved by the FDA in 1998, acquired by Organogenesis from Shire in 2014.
Organogenesis, Inc. & Novartis AG
A bi-layered living skin substitute made from a dermal layer of human cells (fibroblasts) in a bovine type I collagen and an overlying cornified epidermal layer of living human keratinocytes. Apligraf is indicated for the treatment of chronic venous leg ulcers and diabetic foot ulcer. Approved by U.S. FDA in July 1998 and June 2000, respectively.
A dermal substitute used to help in the wound closure of diabetic foot ulcers. It is made from human cells (fibroblasts), placed on a dissolvable mesh material. Organogenesis acquired Dermagraft from Shire in 2014. Approved by U.S. FDA in September 2001.