Zo ziet de markt er volgens Bellicum uit:
Due to its ability to achieve long-term disease remission or functional cure, allogeneic HSCT is standard of care for hematological cancers, such as leukemias and lymphomas, as well as certain nonmalignant blood disorders, such as severe combined immunodeficiency, Wiskott-Aldrich Syndrome, beta thalassemia, sickle cell disease, severe aplastic anemia and Fanconi anemia. However, HSCT is not available to a majority of these patients due to the lack of a fully matched donor. We believe BPX-501 can address this major shortcoming by making haploidentical transplants a safe and effective option.
Within the United States and Europe, there are approximately 60,000 HSCTs performed each year, of which 43% are allogeneic. Within the allogeneic HSCT market, finding a compatible donor is a major limitation to successful treatment. A test comparing the human leukocyte antigen, or HLA types, is used to measure compatibility of the donor and the patient. A sibling with an exactly matching HLA type is an ideal donor for the patient. However, only 30% of potential allogeneic HSCT patients have a perfect match, and this percentage is expected to continue declining as the average family size continues to decrease. A suitable match can be identified in databases of unrelated donors for approximately 75% of patients of white European descent, but a suitable match of patients from other ethnicities is difficult to find and occurs in approximately 20-40% of patients, often not in time to enable the transplant before disease has progressed or the patient has died. This leaves a large pool of patients in need of alternative treatment options.
De totale markt bedraagt derhalve een kleine 26.000 transplantaties. Kiadis heeft het ook over deze aantallen. Alleen Molmed houdt het op een bescheiden 11.000, maar die telt alleen diegenen die aan een vorm van bloedkanker lijdt, ze zitten daarmee 8.000 onder de Kiadis aantallen. Ze hebben het wel over een jaarlijks groeipercentage van 30%. Hun uiteindelijke jaarlijkse aantallen behandelingen van 2500 stuks klinkt dan ook niet als overambitieus. Bellicum heeft zich alvast op de markt van kinderen met leukemie en niet kanker gerelateerde ziektes gestort. Iedereen is dus bezig zich een plaats onder de zon te verwerven.