Table 1.
European orphan designations for non-cancer medicinal products in cancer indications.
| Active substance | Original indication | EU OD indication (cancer) | Designation date | Sponsor |
|---|---|---|---|---|
| Brivudine | Viral infections | Pancreatic cancer | 2010 | RESprotect GmbH |
| Chloroquine | Malaria | Glioma | 2014 | DualTpharma B.V. |
| Eflornithine + Sulindac | African trypanosomiasis, hirsutism | Familial adenomatous polyposis | 2010 | Cancer Prevention Pharma Ltd |
| Neuroblastoma | 2011 | Cancer Prevention Pharma Ltd | ||
| Glioma | 2016 | Orbus Therapeutics Ltd | ||
| Inflammatory conditions | Familial adenomatous polyposis | 2013 | Cancer Prevention Pharma Ltd | |
| Flucytosine | Fungal infections | Glioma | 2018 | Richardson Associates Regulatory Affairs Ltd |
| Itraconazole | Fungal infections | Naevoid basal-cell carcinoma syndrome | 2017 | Mayne Pharma UK Ltd |
| Ketoconazole | Fungal infections | Granulosa cell tumors | 2017 | Grupo Español de Tumores Huérfanos e Infrecuentes (GETHI) |
| Miltefosine | Leishmaniasis | Cutaneous T-cell lymphoma | 2008 | ExperGen Drug Development GmbH |
| Naloxone | Opioid overdose | Cutaneous T-cell lymphoma | 2012 | Winston Laboratories Ltd |
| Propranolol | Hypertension | Soft tissue sarcoma | 2016 | The Anticancer Fund |
| Valproic acid + Carboplatin | Epilepsy | Diffuse large B-cell lymphoma | 2016 | Valcuria AB |
| Multiple cancer types | Glioma | 2018 | Dr Ulrich Granzer | |
| Zoledronic acid | Osteoporosis | Glioma | 2016 | Laboratorio Italiano Biochimico Farmaceutico Lisapharma S.p.A. |
Orphan designation (OD), naloxone hydrochloride dehydrate, naloxone.
Source: Community Register of orphan medicinal products for human use, last update in August 2019.