Background
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.
Overview
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 substance | PIP initially agreed | Children with a melanoma to be investigated | Children with a solid malignant tumour in early trial | Early trial with children started | Children could be included in trial with adults with melanoma that started | Melanoma marketing authorisation in adults submitted on | Marketing authorisation in children on (indication) |
|---|
| bempegaldesleukin | 2019-08-14 | Inclusion possible in solid malignant tumour trial | Yes | 2021-04-19 | 2020-10-14 | None known so far | |
| binimetinib | 2016-03-18 | Yes | Yes (not in PIP) | 2016-04 (ras/raf activated solid malignant tumours) | | 2017-07-28 | |
| dabrafenib | 2012-01-27 | Inclusion possible in solid malignant tumour trial | Yes | 2012-09 (melanoma), 2013-02-27 (brain tumours), 2015-01-15 (V600 mutant cancers) | | 2012-07-24 | |
| encorafenib | 2016-03-18 | Yes | None found so far | None found so far | | 2018-09-19 | |
| ipilimumab | 2011-06-08 | Yes | Yes | 2007-10-01 (solid tumours, lymphoma), 2012-02-17 (melanoma) | | 2010-05-05 | Yes (melanoma, 2018-01-18) |
| mage-a3 recombinant protein | 2012-07-26 | No (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 | |
| nivolumab | 2014-03-07 | Inclusion possible in solid malignant tumour trial | Yes | 2015-02-02 | | 2014-09-02 | |
| paclitaxel | 2013-04-26 | Inclusion possible in solid malignant tumour trial | | | | | No (data evaluated by 2020-01-30, no authorisation in children recommended) |
| pembrolizumab | 2014-03-07 | Inclusion possible in solid malignant tumour trial | Yes | 2015-03-18 | | 2014-06-04 | Yes [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 / nivolumab | 2020-08-14 | No (paediatric development based on modeling, simulation, extrapolation) | No | No | No | None known so far | |
| selumetinib | 2015-07-10 | Extrapolation, subsequently waived | No | 2011-05-11 (plexiform neurofibroma) | | 2021-06-17 | Yes (treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above) |
| talimogene laherparepvec | | Inclusion possible in solid malignant tumour trial | Yes (non CNS) | 2017-08-16 | | 2014-08-28 | |
| trametinib | 2012-02-28 | Inclusion possible in solid malignant tumour trial | Yes | 2015-01-15 | | 2013-02-07 | |
| vemurafenib | 2011-04-08 | Yes, subsequently waived | No | 2013-01 | | 2011-05-04 | |
References
{5011:JSP835FR};{5011:HLPRMN5D} default asc no 452 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