This is a manually curated listing of anti-cancer medicines that are authorised for children with cancer (or with a progressive neoplastic disease). It is limited to those anti-cancer medicines that are centrally authorised in the European Union (see EMA). At this time, no information is provided here on the degree of innovation, on the clinical utility, on treatment guidelines or on how often the medicines are used to treat children with cancer.
- For cancer medicines that are likely authorised for children, see our web app here.
- For FDA-approved medicines for the treatment of paediatric cancers, see this FDA page (83 medicines as of 2024-03-28).
- For trials in children of cancer medicines authorised in adults, see our post (data as of 2022-04-24).
Table last updated 2024-03-28. Not included are specific supportive medicines (such as Pedmarqsi, authorised on 2023-05-26, indication: prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to less than 18 years of age with a localised, non-metastatic, solid tumour).
| # | Active substance | First authorisation | Authorised for children | Indication(s) (excerpt concerning paediatric population) |
|---|---|---|---|---|
| 33 | trametinib (Spexotras) | 2024-01-05 | 2024-01-05 | Low-grade glioma: Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. High-grade glioma: Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment. |
| 32 | dabrafenib | 2023-11-15 | 2023-11-15 | Finlee in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. Finlee in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment. |
| 31 | recombinant Erwinia asparaginase (crisantaspase) | 2023-07-20 | 2023-07-20 | Enrylaze is indicated as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukaemia (ALL) and lymphoblastic lymphoma (LBL) in adult and paediatric patients (1 month and older) who developed hypersensitivity or silent inactivation to E. coli-derived asparaginase. |
| 30 | tabelecleucel | 2022-12-16 | 2022-12-16 | Ebvallo is indicated as monotherapy for treatment of adult and paediatric patients 2 years of age and older with relapsed or refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate. |
| 29 | crizotinib | 2012-10-23 | 2022-10-28 | (excerpt) XALKORI as monotherapy is indicated for: - The treatment of paediatric patients (age ≥6 to <18 years) with relapsed or refractory systemic anaplastic lymphoma kinase (ALK)-positive anaplastic large cell lymphoma (ALCL). - The treatment of paediatric patients (age ≥6 to <18 years) with recurrent or refractory anaplastic lymphoma kinase (ALK)-positive unresectable inflammatory myofibroblastic tumour (IMT). |
| 28 | relatlimab / nivolumab | 2022-09-15 | 2022-09-15 | Opdualag is indicated for the first-line treatment of advanced (unresectable or metastatic) melanoma in adults and adolescents 12 years of age and older with tumour cell PD-L1 expression < 1%. |
| 27 | selumetinib | 2021-06-17 | 2021-06-17 | Koselugo as monotherapy is indicated for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above. |
| 25 | pembrolizumab | 2015-07-17 | 2021-03-09 | (excerpt) KEYTRUDA as monotherapy is indicated for the treatment of adult and paediatric patients aged 3 years and older with relapsed or refractory classical Hodgkin lymphoma who have failed autologous stem cell transplant (ASCT) or following at least two prior therapies when ASCT is not a treatment option. |
| 26 | selpercatinib | 2021-02-11 | 2021-02-11 | (excerpt) Retsevmo as monotherapy is indicated for the treatment of adults and adolescents 12 years and older with advanced RET-mutant medullary thyroid cancer (MTC) who require systemic therapy following prior treatment with cabozantinib and/or vandetanib. |
| 24 | melphalan | 2020-11-16 | 2020-11-16 | High-dose of PHELINUN used alone or in combination with other cytotoxic medicinal products and/or total body irradiation is indicated in the treatment of: - multiple myeloma, - malignant lymphoma (Hodgkin, non-Hodgkin lymphoma), - acute lymphoblastic and myeloblastic leukemia, - childhood neuroblastoma, - ovarian cancer, - mammary adenocarcinoma. PHELINUN in combination with other cytotoxic medicinal products is indicated as reduced intensity conditioning (RIC) treatment prior to allogeneic haematopoietic stem cell transplantation (allo-HSCT) in malignant haematological diseases in adults. PHELINUN in combination with other cytotoxic medicinal products is indicated as conditioning regimen prior to allogeneic haematopoietic stem cell transplantation in haematological diseases in the paediatric population as: - Myeloablative conditioning (MAC) treatment in case of malignant haematological diseases - RIC treatment in case of non-malignant haematological diseases. |
| 23 | entrectinib | 2020-07-31 | 2020-07-31 | Rozlytrek as monotherapy is indicated for the treatment of adult and paediatric patients 12 years of age and older with solid tumours expressing a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, - who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and - who have not received a prior NTRK inhibitor - who have no satisfactory treatment options (see sections 4.4 and 5.1). |
| 22 | rituximab | 1998-06-02 | 2020-03-03 | (excerpt) MabThera in combination with chemotherapy is indicated for the treatment of paediatric patients (aged ≥ 6 months to < 18 years old) with previously untreated advanced stage CD20 positive diffuse large B-cell lymphoma (DLBCL), Burkitt lymphoma (BL)/Burkitt leukaemia (mature B-cell acute leukaemia) (BAL) or Burkitt-like lymphoma (BLL). MabThera, in combination with glucocorticoids, is indicated for the induction of remission in paediatric patients (aged ≥ 2 to < 18 years old) with severe, active GPA (Wegener’s) and MPA. |
| 21 | larotrectinib | 2019-09-19 | 2019-09-19 | VITRAKVI as monotherapy is indicated for the treatment of adult and paediatric patients with solid tumours that display a Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusion, - who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and - who have no satisfactory treatment options (see sections 4.4 and 5.1 of the SmPC). |
| 20 | treosulfan | 2019-06-20 (Trecandi) | 2019-06-20 | Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients with malignant and non-malignant diseases, and in paediatric patients older than one month with malignant diseases. |
| 19 | blinatumomab | 2015-11-22 | 2018-08-23 | BLINCYTO is indicated as monotherapy for the treatment of adults with Philadelphia chromosome negative CD19 positive relapsed or refractory B-precursor acute lymphoblastic leukaemia (ALL). BLINCYTO is indicated as monotherapy for the treatment of paediatric patients aged 1 year or older with Philadelphia chromosome negative CD19 positive B-cell precursor ALL which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic hematopoietic stem cell transplantation. |
| 18 | tisagenlecleucel | 2018-08-22 | 2018-08-22 | Kymriah is indicated for the treatment of: Paediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse. Adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. |
| 17 | dasatinib | 2006-11-20 | 2018-07-02 (CML) 2018-12-13 (ALL) | (excerpt) SPRYCEL is indicated for the treatment of paediatric patients with: - newly diagnosed Ph+ CML in chronic phase (Ph+ CML-CP) or Ph+ CML-CP resistant or intolerant to prior therapy including imatinib. - newly diagnosed Ph+ ALL in combination with chemotherapy |
| 16 | gemtuzumab ozogamicin | 2018-04-18 | 2018-04-18 | MYLOTARG is indicated for combination therapy with daunorubicin (DNR) and cytarabine (AraC) for the treatment of patients age 15 years and above with previously untreated, de novo CD33-positive acute myeloid leukaemia (AML), except acute promyelocytic leukaemia (APL) |
| 15 | ipilimumab | 2011-07-13 | 2018-01-18 | (excerpt) Yervoy is indicated for the treatment of advanced (unresectable or metastatic) melanoma in adults, and adolescents 12 years of age and older. |
| 14 | nilotinib | 2007-11-19 | 2017-11-15 | Tasigna is indicated for the treatment of: -adult and paediatric patients with newly diagnosed Philadelphia chromosome positive chronic myelogenous leukaemia (CML) in the chronic phase, -adult patients with chronic phase and accelerated phase Philadelphia chromosome positive CML with resistance or intolerance to prior therapy including imatinib. Efficacy data in patients with CML in blast crisis are not available, -paediatric patients with chronic phase Philadelphia chromosome positive CML with resistance or intolerance to prior therapy including imatinib. |
| 13 | dinutuximab beta | 2017-05-08 | 2017-05-08 | Qarziba is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilised by other suitable measures. In patients with a history of relapsed/refractory disease and in patients who have not achieved a complete response after first line therapy, Qarziba should be combined with interleukin-2 (IL-2). |
| 12 | methotrexate | 2017-03-28 | 2017-03-28 | (excerpt) Polyarthritic forms of active, severe juvenile idiopathic arthritis (JIA) in adolescents and children aged 3 years and over when the response to non-steroidal anti-inflammatory drugs (NSAIDs) has been inadequate. Maintenance treatment of acute lymphoblastic leukaemia (ALL) in adults, adolescents and children aged 3 years and over. |
| 11 | vandetanib | 2012-02-17 | 2016-12-16 | Caprelsa is indicated for the treatment of aggressive and symptomatic medullary thyroid cancer (MTC) in patients with unresectable locally advanced or metastatic disease. Caprelsa is indicated in adults, children and adolescents aged 5 years and older. For patients in whom re-arranged-during-transfection (RET) mutation is not known or is negative, a possible lower benefit should be taken into account before individual treatment decision. |
| 10 | recombinant asparaginase | 2016-01-14 | 2016-01-14 | Spectrila is indicated as a component of antineoplastic combination therapy for the treatment of acute lymphoblastic leukaemia (ALL) in paediatric patients from birth to 18 years and adults. |
| 9 | pegaspargase | 2016-01-14 | 2016-01-14 | Oncaspar is indicated as a component of antineoplastic combination therapy in acute lymphoblastic leukaemia (ALL) in paediatric patients from birth to 18 years, and adult patients. |
| 8 | mercaptopurine | 2012-08-03 | 2012-08-03 | Xaluprine is indicated for the treatment of acute lymphoblastic leukaemia (ALL) in adults, adolescents and children. |
| 7 | thiotepa | 2010-03-15 | 2010-03-15 | TEPADINA is indicated, in combination with other chemotherapy medicinal products: with or without total body irradiation (TBI), as conditioning treatment prior to allogeneic or autologous haematopoietic progenitor cell transplantation (HPCT) in haematological diseases in adult and paediatric patients; when high dose chemotherapy with HPCT support is appropriate for the treatment of solid tumours in adult and paediatric patients.". It is proposed that Tepadina must be prescribed by physicians experienced in conditioning treatment prior to haematopoietic progenitor cell transplantation. |
| 6 | mifamurtide | 2009-06-03 | 2009-06-03 | Mepact is indicated in children, adolescents and young adults for the treatment of high-grade resectable non-metastatic osteosarcoma after macroscopically complete surgical resection. It is used in combination with post-operative multi-agent chemotherapy. Safety and efficacy have been assessed in studies of patients 2 to 30 years of age at initial diagnosis (see section 5.1). |
| 5 | nelarabine | 2007-08-22 | 2007-08-22 | Nelarabine is indicated for the treatment of patients with T-cell acute lymphoblastic leukaemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens. Due to the small patient populations in these disease settings, the information to support these indications is based on limited data. |
| 4 | imatinib | 2001-11-07 | 2006-09-13 (CML) 2013-06-27 (ALL) | (excerpt) Glivec is indicated for the treatment of • adult and paediatric patients with newly diagnosed Philadelphia chromosome (bcr-abl) positive (Ph+) chronic myeloid leukaemia (CML) for whom bone marrow transplantation is not considered as the first line of treatment. • adult and paediatric patients with Ph+ CML in chronic phase after failure of interferon-alpha therapy, or in accelerated phase or blast crisis. • adult and paediatric patients with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) integrated with chemotherapy. The effect of Glivec on the outcome of bone marrow transplantation has not been determined. In adult and paediatric patients, the effectiveness of Glivec is based on overall haematological and cytogenetic response rates and progression-free survival in CML, on haematological and cytogenetic response rates in Ph+ ALL, MDS/MPD, on haematological response rates in HES/CEL and on objective response rates in adult patients with unresectable and/or metastatic GIST and DFSP and on recurrence-free survival in adjuvant GIST. The experience with Glivec in patients with MDS/MPD associated with PDGFR gene re-arrangements is very limited (see section 5.1). Except in newly diagnosed chronic phase CML, there are no controlled trials demonstrating a clinical benefit or increased survival for these diseases. |
| 3 | clofarabine | 2006-05-29 | 2006-05-29 | Treatment of acute lymphoblastic leukaemia (ALL) in paediatric patients who have relapsed or are refractory after receiving at least two prior regimens and where there is no other treatment option anticipated to result in a durable response. Safety and efficacy have been assessed in studies of patients ≤ 21 years old at initial diagnosis (see section 5.1). |
| 2 | busulfan | 2003-07-09 | 2005-07-27 | (excerpt) Busilvex followed by cyclophosphamide (BuCy4) or melphalan (BuMel) is indicated as conditioning treatment prior to conventional haematopoietic progenitor cell transplantation in paediatric patients. |
| 1 | temozolomide | 1999-01-26 | 1999-01-26 | (excerpt) Temodal is indicated for the treatment of: - children from the age of three years, adolescents and adult patients with malignant glioma, such as glioblastoma multiforme or anaplastic astrocytoma, showing recurrence or progression after standard therapy. |