Table 1:
Targeted therapies approved by the FDA and/or EMA for pediatric cancer
| Year Approved | |||||
|---|---|---|---|---|---|
| Drug | Pediatric Indication | FDA | EMA | Pediatric Phase 1* Response Rate in Targeted Population | Number of Patients |
| Tretinoin | Acute promyelocytic leukemia (APML) | 1995 | NC | 83% (all CR)(101) | 6 |
| Everolimus | Subependymal giant cell astrocytoma (SEGA) | 2010 | 2011 | 75%(102) | 28 |
| Dinutuximab | Neuroblastoma | 2015 | 2015** | 66%(103) | 6 |
| Blinatumomab | Relapsed/refractory (R/R) B acute lymphoblastic leukemia (ALL) | 2016 | 2015 | 30% (all minimal residual disease negative)(21) | 23 |
| Pembrolizumab | R/R classic Hodgkin Lymphoma, primary mediastinal B-cell lymphoma, Merkel cell carcinoma, MSI-high tumors | 2017 | N/A | 60% (Hodgkin)(66) | 15 |
| Ipilimumab | Melanoma (≥12 years) | 2017 | 2017 | 0(67) | 12 |
| Gemtuzumab | R/R acute myeloid leukemia (AML) | 2017 | 2018*** | 28%(104) | 29 |
| Tisagenlecleucel | R/R B-ALL | 2017 | 2018 | 90%(105) | 30 |
| Dasatinib | CML / Ph+ ALL | 2017 | 2017 | 82% CCyR (CML chronic phase [CP])(22) | 17 |
| Imatinib | Ph+ ALL and chronic myeloid leukemia (CML) | 2017 | 2013 | 70% ALL, 83% CML CP(23) | 10 ALL, 14 CML |
| Nilotinib | CML | 2018 | 2017 | 90%(24) | 10 |
| Larotrectinib | TRK fusion solid tumors | 2018 | 2019 | 93%(13) | 15 |
| Entrectinib | TRK fusion solid tumors (≥12 years) | 2019 | 2020 | 100%(26) | 6 |
| Tazemetostat | Epithelioid sarcoma (≥16 years) | 2020 | N/A | 29%(106) | 7 |
| Selumetinib | Plexiform neurofibroma | 2020 | N/A | 71%(107) | 24 |
| Selpercatinib | RET-mutant/fusion thyroid cancer (≥12 years) | 2020 | N/A | Not yet reported | |
| Rituximab | Mature B-cell lymphomas | N/A | 2020 | 41%(108) | 87 |
*
if no pediatric phase 1 in targeted population, first published phase 2 response rate
**
Dinutuximab was approved in 2015, but authorization for commercialization was then withdrawn by the company. Dinutuximab beta was approved in 2017
***:
Only children ≥ 15 years old
NC: no centralised procedure when approved (approved by each individual EU country)
N/A: not approved