Development going beyond rare adult tumour


This is a selection from all paediatric investigation plans in the therapeutic area of oncology, such as listed in this webapp or made public by the EMA. Melanoma is malignant neoplasm that is rare in adults and during childhood . The development of anti-cancer medicines for adults with malignant melanoma triggered the need to discuss a paediatric development, which for some medicines was limited to melanoma and for others went to include also other malignant tumours, likely in view of the medicine's pharmacological action. Some medicines listed below are listed as "voluntary" PIPs in . Eventually, this development model may lead to new medicines for cancers primarily affecting children.


The table is manually curated to include paediatric investigation plans that refer to melanoma or solid malignant tumours and where melanoma was the first authorised indication. Information on trial start was taken from ClinicalTrials.Gov. The date of submission of marketing authorisation was taken from the European Public Assessment Report (EPAR). Last update: 2021-12-30.
Active substancePIP initially agreedChildren with a melanoma to be investigatedChildren with a solid malignant tumour in early trialEarly trial with children startedChildren could be included in trial with adults with melanoma that startedMelanoma marketing authorisation in adults submitted onMarketing authorisation in children on (indication)
bempegaldesleukin2019-08-14Inclusion possible in solid malignant tumour trialYes2021-04-192020-10-14None known so far
binimetinib2016-03-18YesYes (not in PIP)2016-04 (ras/raf activated solid malignant tumours)2017-07-28
dabrafenib2012-01-27Inclusion possible in solid malignant tumour trialYes2012-09 (melanoma), 2013-02-27 (brain tumours), 2015-01-15 (V600 mutant cancers)2012-07-24
encorafenib2016-03-18YesNone found so farNone found so far2018-09-19
ipilimumab2011-06-08YesYes2007-10-01 (solid tumours, lymphoma), 2012-02-17 (melanoma)2010-05-05Yes (melanoma, 2018-01-18)
mage-a3 recombinant protein2012-07-26No (waived 2014-10-30 "on the grounds that the specific medicinal product is likely to be ineffective or unsafe in part or all of the paediatric population")None known so far
nivolumab2014-03-07Inclusion possible in solid malignant tumour trialYes2015-02-022014-09-02
paclitaxel2013-04-26Inclusion possible in solid malignant tumour trialNo (data evaluated by 2020-01-30, no authorisation in children recommended)
pembrolizumab2014-03-07Inclusion possible in solid malignant tumour trialYes2015-03-182014-06-04Yes [classical Hodgkin lymphoma (cHL) 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]
relatlimab / nivolumab2020-08-14No (paediatric development based on modeling, simulation, extrapolation)NoNoNoNone known so far
selumetinib2015-07-10Extrapolation, subsequently waivedNo2011-05-11 (plexiform neurofibroma)2021-06-17Yes (treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above)
talimogene laherparepvecInclusion possible in solid malignant tumour trialYes (non CNS)2017-08-162014-08-28
trametinib2012-02-28Inclusion possible in solid malignant tumour trialYes2015-01-152013-02-07
vemurafenib2011-04-08Yes, subsequently waivedNo2013-012011-05-04


Brecht Ines B., Paoli Angela, Bisogno Gianni, Orbach Daniel, Schneider Dominik T., Leiter Ulrike, Offenmueller Sonja, et al. 2018. “Pediatric Patients with Cutaneous Melanoma: A European Study.” Pediatric Blood & Cancer 0 (0).
European Medicines Agency. 2017. “10-Year Report to the European Commission - General Report on the Experience Acquired as a Result of the Application of the Paediatric Regulation.”

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