ATMP success stories
ATMPs are already saving and improving lives but there is much more to come for patients in need!
There are cell and gene therapies being developed all over the world for a variety of indications.
You can see a summary of products currently approved around the world in the ISCT annual report here.
Success has been seen around the world with life saving treatments or treatments that mean patients can live a normal life. For diseases like ADA/SCID, where the patients immune system is so compromised any infection could kill them, introduction of gene corrected cells has allowed these children to live normal lives. In cancer we have seen great advanced in CAR-T treatments where introduced cells stimulate the body to fight and remove cancers using its own immune system. We are really excited to be involved in development of this field to bring new revolutionary treatments to patients.
In Europe gene and cell therapies are classified as ATMPs.
The following is a list of EMA (European Medicines Agency) approved ATMPs.
Alofisel maintained long-term remission of treatment refractory complex perianal fistulas in patients with Crohn’s disease over 52 weeks. It is a donor fat derived mesenchymal stem cell treatment for perianal fistula in Chrohn’s disease. Classified as a somatic cell therapy (sCTMP) it is sold by Takeda Pharma.
Holocar is based on autologous limbal stem cells and capable to restore the eyesight of patients with severe cornea damage. Sold be Chiesi Farmaceutici the patient derived corneal epithelial limbal stem cell are grown in the lab and returned to patients with severe limbal stem cell deficiency. The Product is classified as a somatic cell therapy (sCTMP)
Imlygic significantly Increased Durable Response Rates in Patients With Unresectable Melanoma That is Regionally or Distantly Metastatic. Imlygic is a gene therapy medicinal Product (GTMP) sold by Amgen Europe B.V. and is a modified herpes simplex virus containing the GM-CSF gene against metastatic melanoma.
Spherox has been shown to be effective at treating defects in the knee cartilage between 1 and 10 cm2 in size. The Product is sold by CO.DON AG and contains patient derived chondrocytes that are grown in the lab and then transplanted to the patients damaged cartilaged. This Product is classified as a Tissue Engineered Product (TEP).
Strimvelis gives patients a working gene of ADA in their blood stem cells improving their ability to fight infection. The effects are expected to last for the patient’s lifetime.. The Product is sold by Orchard Therapeutics (Netherlands) BV and involves retrovirus modification of a patients blood stem cells in the lab Before being returned to the patient. Strimvelis is classified as a gene therapy medicinal Product (GTMP).
Yescarta is made from your own white blood cells, which have been modified to recognize and attack your lymphoma cells. It is the first CAR-T therapy for adults living with certain types of non-Hodgkin lymphoma, who have failed at least 2 other kinds of treatment. The Product is sold by Kite Pharma EU B.V. and is classified as a gene therapy medicinal Product (GTMP).
Zalmoxis, When given after bone marrow transplantation, helps the patient to build up their immune system and so helps to protect them from infections. The product is sold by MolMed ApA and involves genetic modification of donor White blood cells with delta LNGFR, thus is classified as a gene therapy medicinal Product (GTMP).
Disease success but commercial failure;
Glybera provided a one-off solution by introducing copies of the relevant gene to produce the deficient lipase indefinitely; the longest term study has proven its efficacy for at least six years. Glybera was sold by uniQure biopharma and based on an adeno-associated virus with the LPL (S447X) gene for familial lipoprotein lipase deficiency.