Table 3.

Status of new oncology drugs approved by the EMA since 2007 with regard to their development in children and adolescent

Common nameMedicine nameMarketing authorization holderYearIndication in adultsApproved for use in childrenPIPPediatric indication in the PIPPublished waiverCommentIs the mechanism of action potentially relevant for pediatric malignancies?
EverolimusVotubiaNovartis Europharm Ltd.201111SEGA associated with tuberous sclerosis complex0Drug authorized for treatment of patients ages 3 years and older; authorization was based on the completion of a PIP1
NelarabineAtrianceGlaxo Group Ltd.200710T-cell ALL and T-cell lymphoblastic lymphoma0Drug indicated in children for the treatment of the same condition1
ThiotepaTepadinaAdienne Srl201010HPCT in hematologic diseases and solid tumors in adult and pediatric patients0Drug indicated in children for the treatment of hematologic malignancies and malignant solid tumors1
6-Mercaptopurine monohydrateXaluprineNova Laboratories Ltd.201210ALL in adults, adolescents and children.0First oral suspension of 6-mercaptopurine1
IpilimumabYervoyBristol-Myers Squibb Pharma EEIG2011Advanced melanoma02Melanoma; solid tumors01
EverolimusAfinitorNovartis Europharm Ltd.2009Neuroendocrine tumors and renal cell carcinoma01SEGA associated with tuberous sclerosis complex2Waiver in neuroendocrine tumors and renal cell carcinoma; a PIP was approved for SEGA1
NilotinibTasignaNovartis Europharm Ltd.2007Philadelphia-chromosome-positive chronic myelogenous leukemia01Chronic myelogenous leukemia01
PlerixaforMozobilGenzyme Europe B.V.2009Mobilization of hematopoietic stem cells01Mobilization of hematopoietic stem cells01
PazopanibVotrientGlaxo Group Ltd.2010Renal cell carcinoma01Soft tissue sarcomas0A class waiver was issued for renal cancer. Then a PIP was approved when the drug was developed in adult sarcomas1
Pixantrone dimaleatePixuvriCTI Life Sciences Ltd.2012Non-Hodgkin B-cell lymphoma01NHL leukemia01
VemurafenibZelborafRoche Registration Ltd.2012BRAF V600 mutation-positive unresectable or metastatic melanoma01BRAF V600 mutation-positive metastatic melanoma0Pediatric tumors other than melanoma have B-RAF mutations1
VandetanibCaprelsaAstraZeneca AB2012Thyroid cancer01Thyroid cancerVandetanib targets are altered in pediatric malignancies other than thyroid cancer1
LenalidomideRevlimidCelgene Europe Ltd.2007Multiple myeloma002Immunomodulating agent; ongoing pediatric development1
TemsirolimusToriselPfizer Ltd.2007Renal-cell carcinoma001mTOR is a relevant target in pediatric malignancies; ongoing pediatric development1
LapatinibTyverbGlaxo Group Ltd.2008HER2-positive (ErbB2) breast cancer (ErbB2):001Inhibitor of EGFR and HER2-neu receptors1
PanitumumabVectibixAmgen Europe B.V.2007Wild-type KRAS metastatic colorectal cancer000Monoclonal antibody inhibiting the EGFR TK receptor1
TrabectedinYondelisPharma Mar S.A.2007Advanced soft tissue sarcoma000Cytotoxic compound to be evaluated in children; ongoing development in children with solid malignancies1
Nab-paclitaxelAbraxaneCelgene Europe Ltd.2008Metastatic breast cancer000Cytotoxic compound that proved to be more active than paclitaxel1
ThalidomideThalidomide CelgeneCelgene Europe Ltd.2008Multiple myeloma000Immunomodulating agent1
AzacitidineVidazaCelgene Europe Ltd.2008Myelodysplastic syndromes, chronic myelomonocytic leukemia, acute myeloid leukemia000Ongoing development in children with leukemias1
VinflunineJavlorPierre Fabre MĂ©dicament2009Transitional cell carcinoma of the urothelial tract000Vinca-alkaloids are a major class of drugs in the treatment of several pediatric malignancies1
GefitinibIressaAstraZeneca AB2009Non–small cell lung carcinoma with activating mutations of EGFR-TK000Ongoing development in children with solid tumors1
OfatumumabArzerraGlaxo Group Ltd2010Chronic lymphocytic leukemia000An anti-CD20 monoclonal antibody; CD20 is expressed on B lymphocytes and B-cell tumors (CLL and NHL)1
CabazitaxelJevtanaSanofi-Aventis group2011Hormone-refractroy metastatic prostate cancer000A taxoid that crosses blood brain barrier. Studies in children are warranted1
Eribulin mesylateHalavenEisai Europe Ltd.2011Metastatic breast cancer000Tubulin-based antimitotic drug1
AxitinibInlytaPfizer Ltd.2012Renal cell carcinoma000Angiogenesis is a major therapeutic target in pediatric malignancies as well1
Tegafur/gimeracil/oteracilTeysunoNordic Group BV2011Advanced gastric cancer0005-Fluorouracil did not show antitumor activity in pediatric malignancies0
Abiraterone acetateZytigaJanssen-Cilag International N.V.2011Metastatic castration-resistant prostate cancer000Androgen biosynthesis inhibitor; mechanism of action is not relevant for pediatric malignancies0

Abbreviations: EGFR, EGF receptor; HPCT, hematopoietic progenitor cell transplantation; NHL, non-Hodgkin lymphoma; SEGA, subependymal giant cell astrocytoma; TK, tyrosine kinase.