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 of the medicines listed below are listed as "voluntary" PIPs in . Eventually, this development model may lead to new medicines for paediatric-specific cancers.


The table includes 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: 2018-04-25.
Active substanceChildren with a melanoma investigatedChildren with a solid malignant tumour in early trialEarly trial with children startedMelanoma marketing authorisation in adults submitted onMarketing authorisation in children on (indication)
selumetinibExtrapolation, subsequently waivedNo2011-05-11 (plexiform neurofibroma)None known so far
dabrafenibInclusion possible in solid malignant tumour trialYes2012-09 (melanoma), 2013-02-27 (brain tumours), 2015-01-15 (V600 mutant cancers)2012-07-24
vemurafenibYes, subsequently waivedNo2013-012011-05-04
ipilimumabYesYes2007-10-01 (solid tumours, lymphoma), 2012-02-17 (melanoma)2010-05-05Yes (melanoma, 2018-01-18)
binimetinibYesYes (not in PIP)2016-04 (ras/raf activated solid malignant tumours)None known so far
encorafenibYesNone found so farNone found so farNone known so far
trametinibInclusion possible in solid malignant tumour trialYes2015-01-152013-02-07
nivolumabInclusion possible in solid malignant tumour trialYes2015-02-022014-09-02
pembrolizumabInclusion possible in solid malignant tumour trialYes2015-03-182014-06-04
talimogene laherparepvecInclusion possible in solid malignant tumour trialYes (non CNS)2017-08-162014-08-28


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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