Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities (2024)
Chapter: Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022
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Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Brand name | Generic Name | Approved by EMA | EMA Approved as Orphan | EMA Approval Year | Approved by FDA | FDA Approved as Orphan | FDA Approval Year | Divergent Decision | Therapeutic Area | Reason for non-approval in EMA | Reason for non-approval in FDA |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Abecma | idecabtagene vicleucel | Y | Y | 2021 | Y | Y | 2021 | N | L01 | ||
| Adakveo | crizanlizumab | Y | Y | 2020 | Y | Y | 2019 | N | B06 | ||
| Alofisel | darvadstrocel | Y | Y | 2018 | N | N | #N/A | Y | L04 | Not submitted | |
| Amondys 45 | casimersen | N | N | #N/A | Y | Y | 2021 | Y | M09 | Not submitted | |
| Amvuttra | vutrisiran sodium | Y | Y | 2022 | Y | Y | 2022 | N | N07 | ||
| Andexxa | coagulation factor Xa [recombinant], inactivated-zhzo | Y | N | 2019 | Y | Y | 2018 | N | B02 | ||
| ANTHIM | obiltoxaximab | Y | Y | 2020 | Y | Ya | 2016 | N | J01 | ||
| Artesunate Amivas | Artesunate | Y | Y | 2021 | Y | Y | 2020 | N | P01 | ||
| Asparlas | calaspargase pegol-mknl | N | N | #N/A | Y | Y | 2018 | Y | L01 | Not submitted | |
| Ayvakit | Avapritinib | Y | Y | 2020 | Y | Y | 2020 | N | L01 | ||
| Besremi | ropeginterferon alfa-2b | Y | N | 2019 | Y | Y | 2021 | N | L03 | ||
| Blenrep | belantamab mafodotin | Y | Y | 2020 | Y | Y | 2020 | N | L01 | ||
| Braftovi | encorafenib | Y | N | 2018 | Y | Y | 2018 | N | L01 |
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Breyanzi | lisocabtagene maraleucel | Y | N | 2022 | Y | Y | 2021 | N | L01 | ||
| Brukinsa | zanubrutinib | Y | N | 2021 | Y | Y | 2019 | N | L01 | ||
| Bylvay | odevixibat | Y | Y | 2021 | Y | Y | 2021 | N | A05 | ||
| Cablivi | caplacizumab | Y | Y | 2018 | Y | Y | 2019 | N | B01 | ||
| Camzyos | mavacamten | Y | N | 2023 | Y | Y | 2022 | N | C01 | ||
| Carvykti | ciltacabtagene autoleucel | Y | Y | 2022 | Y | Y | 2022 | N | L01 | ||
| Copiktra | duvelisib | Y | N | 2021 | Y | Y | 2018 | N | L01 | ||
| Crysvita | burosumab | Y | Y | 2018 | Y | Y | 2018 | N | M05 | ||
| Cytalux | pafolacianine sodium | N | N | #N/A | Y | Y | 2021 | Y | V04 | Not submitted | |
| Danyelza | naxitamab-gqgk | N | N | #N/A | Y | Y | 2020 | Y | L01 | Not submitted | |
| Daurismo | glasdegib | Y | Y | 2020 | Y | Y | 2018 | N | L01 | ||
| Detectnet | copper Cu 64 dotatate injection | N | N | #N/A | Y | Y | 2020 | Y | V09 | Not submitted | |
| Diacomit | stiripentol | Y | Ya | 2007 | Y | Y | 2018 | N | N03 | ||
| Dojolvi | triheptanoin | N | N | #N/A | Y | Y | 2020 | Y | A16 | Not submitted | |
| Ebanga | ansuvimab-zykl | N | N | #N/A | Y | Y | 2020 | Y | J05 | Not submitted | |
| Ebvallo | Tabelecleucel | Y | Y | 2022 | N | N | #N/A | Y | L01 | Not submitted |
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Brand name | Generic Name | Approved by EMA | EMA Approved as Orphan | EMA Approval Year | Approved by FDA | FDA Approved as Orphan | FDA Approval Year | Divergent Decision | Therapeutic Area | Reason for non-approval in EMA | Reason for non-approval in FDA |
|---|---|---|---|---|---|---|---|---|---|---|---|
| EGATEN | triclabendazole | N | N | #N/A | Y | Y | 2019 | Y | P02 | Not submitted | |
| Elahere | mirvetuximab soravtansine-gynx | N | N | #N/A | Y | Y | 2022 | Y | L01 | Still in review | |
| Elzonris | tagraxofusp | Y | Y | 2021 | Y | Y | 2018 | N | L01 | ||
| Empaveli | pegcetacoplan | Y | Y | 2021 | Y | Y | 2021 | N | L04 | ||
| Enjaymo | sutimlimab | Y | Y | 2022 | Y | Y | 2022 | N | L04 | ||
| Enspryng | satralizumab | Y | Y | 2021 | Y | Y | 2020 | N | L04 | ||
| Epidiolex | cannabidiol | Y | Ya | 2019 | Y | Y | 2018 | N | N03 | ||
| Evkeeza | evinacumab | Y | N | 2021 | Y | Y | 2021 | N | C10 | ||
| Evrysdi | risdiplam | Y | Y | 2021 | Y | Y | 2020 | N | M09 | ||
| Exkivity | mobocertinib succinate | N | N | #N/A | Y | Y | 2021 | Y | L01 | Withdrawn by the sponsor | |
| Fexinidazole | fexinidazole | N | N | #N/A | Y | Y | 2021 | Ya | P01 | Not submitted | |
| Firdapse | amifampridine | Y | Ya | 2009 | Y | Y | 2018 | N | N07 | ||
| Galafold | migalastat hydrochloride | Y | Ya | 2016 | Y | Y | 2018 | N | A16 | ||
| Gamifant | emapalumab-lzsg | N | N | #N/A | Y | Y | 2018 | Y | L04 | Refused | |
| Gavreto | pralsetinib | Y | N | 2021 | Y | Y | 2020 | N | L01 |
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Givlaari | givosiran | Y | Y | 2020 | Y | Y | 2019 | N | A16 | ||
| Hemgenix | etranacogene dezaparvovec-drlb | Y | Ya | 2023 | Y | Y | 2022 | N | B02 | ||
| Hepcludex | bulevirtide | Y | Y | 2020 | N | N | #N/A | Y | J05 | Still in review | |
| Idefirix | imlifidase | Y | Y | 2020 | N | N | #N/A | Y | L04 | Not submitted | |
| Imcivree | setmelanotide | Y | Y | 2021 | Y | Y | 2020 | N | A08 | ||
| Imjudo | tremelimumabactl | Y | N | 2023 | Y | Y | 2022 | N | L01 | ||
| Inmazeb | atoltivimab, maftivimab, and odesivimab-ebgn | N | N | #N/A | Y | Y | 2020 | Y | J05 | Not submitted | |
| Inqovi | decitabine and cedazuridine | Y | N | 2023 | Y | Y | 2020 | N | L01 | ||
| Inrebic | fedratinib | Y | Y | 2021 | Y | Y | 2019 | N | L01 | ||
| Isturisa | osilodrostat | Y | Y | 2020 | Y | Y | 2020 | N | H02 | ||
| Kimmtrak | tebentafusp | Y | Y | 2022 | Y | Y | 2022 | N | L01 | ||
| Koselugo | selumetinib | Y | Y | 2021 | Y | Y | 2020 | N | L01 | ||
| Krazati | adagrasib | Y | N | 2024 | Y | Y | 2022 | N | L01 | ||
| Krintafel | tafenoquine | N | N | #N/A | Y | Y | 2018 | Y | P01 | Not submitted | |
| Kymriah | tisagenlecleucel | Y | Y | 2018 | Y | Ya | 2017 | N | L01 | ||
| Lampit | nifurtimox | N | N | #N/A | Y | Y | 2020 | Y | P01 | Not submitted | |
| Lamzede | velmanase alfa | Y | Y | 2018 | Y | Ya | 2023 | N | A16 |
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Brand name | Generic Name | Approved by EMA | EMA Approved as Orphan | EMA Approval Year | Approved by FDA | FDA Approved as Orphan | FDA Approval Year | Divergent Decision | Therapeutic Area | Reason for non-approval in EMA | Reason for non-approval in FDA |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Libmeldy | Autologous CD34+ cells encoding ARSA gene | Y | Y | 2020 | Y | Y | 2024 | N | N07 | ||
| Livmarli | maralixibat chloride | Y | Y | 2022 | Y | Y | 2021 | N | A05 | ||
| Livtencity | maribavir | Y | Y | 2022 | Y | Y | 2021 | N | J05 | ||
| Lorbrena | lorlatinib | Y | N | 2019 | Y | Y | 2018 | N | L01 | ||
| Lumakras | sotorasib | Y | N | 2022 | Y | Y | 2021 | N | L01 | ||
| Lumoxiti | moxetumomab pasudotox-tdfk | N | N | #N/A | Y | Y | 2018 | Y | L01 | Withdrawn by the sponsor | |
| Lunsumio | Mosunetuzumab | Y | Y | 2022 | Y | Y | 2022 | N | L01 | ||
| Lutathera | lutetium (177Lu) oxodotreotide | Y | Ya | 2017 | Y | Y | 2018 | N | V10 | ||
| Luxturna | voretigene neparvovec | Y | Y | 2018 | Y | Ya | 2017 | N | S01 | ||
| Lytgobi | futibatinib | Y | N | 2023 | Y | Y | 2022 | N | L01 | ||
| Mektovi | binimetinib | Y | N | 2018 | Y | Y | 2018 | N | L01 | ||
| Mepsevii | vestronidase alfa | Y | Y | 2018 | Y | Ya | 2017 | N | A16 | ||
| Monjuvi | tafasitamab-cxix | Y | Y | 2021 | Y | Y | 2020 | N | L01 | ||
| Moxidectin | moxidectin | N | N | #N/A | Y | Y | 2018 | Y | P02 | Not submitted |
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| MYALEPT (BLA #125390) | METRELEPTIN | Y | Y | 2018 | Y | Ya | 2014 | N | A16 | ||
| Mylotarg | gemtuzumab ozogamicin | Y | Y | 2018 | Y | Ya | 2000 | N | L01 | ||
| Nexviazyme | avalglucosidase alfa-ngpt | Y | N | 2022 | Y | Y | 2021 | N | A16 | ||
| Ngenla | somatrogon | Y | Y | 2022 | Y | Ya | 2023 | N | H01 | ||
| Nulibry | fosdenopterin hydrobromide dihydrate | Y | Y | 2022 | Y | Y | 2021 | N | A16 | ||
| Omegaven | fish oil triglycerides | N | N | #N/A | Y | Y | 2018 | Y | B05 | Not submitted | |
| Onpattro | patisiran sodium | Y | Y | 2018 | Y | Y | 2018 | N | N07 | ||
| Opdualag | nivolumab, relatlimab | Y | N | 2022 | Y | Y | 2022 | N | L01 | ||
| Orladeyo | berotralstat | Y | N | 2021 | Y | Y | 2020 | N | B06 | ||
| Oxbryta | voxelotor | Y | Y | 2022 | Y | Y | 2019 | N | B06 | ||
| Oxervate | recombinant human nerve growth factor (rhNGF) | Y | Ya | 2017 | Y | Y | 2018 | N | S01 | ||
| Oxlumo | lumasiran | Y | Y | 2020 | Y | Y | 2020 | N | A16 | ||
| Palynziq | pegvaliase | Y | Y | 2019 | Y | Y | 2018 | N | A16 | ||
| Pemazyre | pemigatinib | Y | Y | 2021 | Y | Y | 2020 | N | L01 |
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Brand name | Generic Name | Approved by EMA | EMA Approved as Orphan | EMA Approval Year | Approved by FDA | FDA Approved as Orphan | FDA Approval Year | Divergent Decision | Therapeutic Area | Reason for non-approval in EMA | Reason for non-approval in FDA |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Pepaxto | melphalan flufenamide hydrochloride | Y | Ya | 2022 | Y | Y | 2021 | N | L01 | ||
| Polivy | polatuzumab vedotin | Y | Y | 2020 | Y | Y | 2019 | N | L01 | ||
| Poteligeo | mogamulizumab | Y | Y | 2018 | Y | Y | 2018 | N | L01 | ||
| Dovprela | pretomanid | Y | Y | 2020 | Y | Y | 2019 | N | J04 | ||
| Prevymis | letermovir | Y | Y | 2018 | Y | Ya | 2017 | N | J05 | ||
| Pyrukynd | mitapivat sulfate | Y | Y | 2022 | Y | Y | 2022 | N | B06 | ||
| Qinlock | ripretinib | Y | Y | 2021 | Y | Y | 2020 | N | L01 | ||
| Reblozyl | luspatercept | Y | Y | 2020 | Y | Y | 2019 | N | B03 | ||
| Relyvrio | sodium phenylbutyrate and taurursodiol | N | N | #N/A | Y | Y | 2022 | Y | N07 | Refused | |
| Retevmo | selpercatinib | Y | N | 2021 | Y | Y | 2020 | N | L01 | ||
| Revcovi | elapegademaselvlr | N | N | #N/A | Y | Y | 2018 | Y | L03 | Not submitted | |
| Rezlidhia | olutasidenib | N | N | #N/A | Y | Y | 2022 | Y | L01 | Not submitted | |
| Rezurock | belumosudil mesylate | N | N | #N/A | Y | Y | 2021 | Y | L04 | Not submitted | |
| Roctavian | valoctocogene roxaparvovec | Y | Y | 2022 | Y | Ya | 2023 | N | B02 |
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Rozlytrek | entrectinib | Y | N | 2020 | Y | Y | 2019 | N | L01 | ||
| Rubraca | rucaparib | Y | Y | 2018 | Y | Ya | 2016 | N | L01 | ||
| Rylaze | asparaginase erwinia chrysanthemi (recombinant)rywn | Y | N | 2023 | Y | Y | 2021 | N | L01 | ||
| Sarclisa | isatuximab | Y | N | 2020 | Y | Y | 2020 | N | L01 | ||
| Scemblix | asciminib hydrochloride | Y | Y | 2022 | Y | Y | 2021 | N | L01 | ||
| Scenesse | afamelanotide | Y | Ya | 2014 | Y | Y | 2019 | N | D02 | ||
| Skysona | elivaldogene autotemcel | Y | Y | 2021 | Y | Y | 2022 | N | A16 | ||
| Skytrofa | lonapegsomatropin | Y | Y | 2022 | Y | Y | 2021 | N | H01 | ||
| Sogroya | somapacitan | Y | Y | 2021 | Y | N | 2020 | N | H01 | ||
| Spevigo | spesolimab | Y | N | 2022 | Y | Y | 2022 | N | L04 | ||
| Sunosi | solriamfetol | Y | N | 2020 | Y | Y | 2019 | N | N06 | ||
| Symdeko | tezacaftor; ivacaftor | Y | Y | 2018 | Y | Y | 2018 | N | R07 | ||
| Tabrecta | capmatinib dihydrochloride monohydrate | Y | N | 2022 | Y | Y | 2020 | N | L01 | ||
| Takhzyro | lanadelumab | Y | Y | 2018 | Y | Y | 2018 | N | B06 | ||
| Tavalisse | fostamatinib | Y | N | 2020 | Y | Y | 2018 | N | B02 | ||
| Tavneos | avacopan | Y | Y | 2022 | Y | Y | 2021 | N | L04 |
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Brand name | Generic Name | Approved by EMA | EMA Approved as Orphan | EMA Approval Year | Approved by FDA | FDA Approved as Orphan | FDA Approval Year | Divergent Decision | Therapeutic Area | Reason for non-approval in EMA | Reason for non-approval in FDA |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Tazverik | tazemetostat | N | N | #N/A | Y | Y | 2020 | Y | L01 | Not submitted | |
| Tecartus | brexucabtagene autoleucel | Y | Y | 2020 | Y | Y | 2020 | N | L01 | ||
| Tecvayli | teclistamab | Y | N | 2022 | Y | Y | 2022 | N | L01 | ||
| Tegsedi | inotersen sodium | Y | Y | 2018 | Y | Y | 2018 | N | N07 | ||
| Tepezza | teprotumumabtrbw | N | N | #N/A | Y | Y | 2020 | Y | L04 | Not submitted | |
| Tepmetko | tepotinib hydrochloride monohydrate | Y | N | 2022 | Y | Y | 2021 | N | L01 | ||
| Terlivaz | terlipressin | N | N | #N/A | Y | Y | 2022 | Y | H01 | Not submitted | |
| Tibsovo | ivosidenib | Y | Ya | 2023 | Y | Y | 2018 | N | L01 | ||
| TPOXX | tecovirimat | Y | N | 2022 | Y | Y | 2018 | N | J05 | ||
| TRIKAFTA (copackaged) | elexacaftor, ivacaftor, tezacaftor | Y | Y | 2020 | Y | Y | 2019 | N | R07 | ||
| Trogarzo | ibalizumab | Y | N | 2019 | Y | Y | 2018 | N | J05 | ||
| Truseltiq | infigratinib phosphate | N | N | #N/A | Y | Y | 2021 | Y | L01 | Withdrawn by the sponsor | |
| Tukysa | tucatinib | Y | N | 2021 | Y | Y | 2020 | N | L01 | ||
| TURALIO | pexidartinib hydrochloride | N | N | #N/A | Y | Y | 2019 | Y | L01 | Refused |
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Ukoniq | umbralisib tosylate | N | N | #N/A | Y | Y | 2021 | Y | L01 | Not submitted | |
| Ultomiris | ravulizumab | Y | N | 2019 | Y | Y | 2018 | N | L04 | ||
| Uplizna | Inebilizumab | Y | N | 2022 | Y | Y | 2020 | N | L04 | ||
| Upstaza | eladocagene exuparvovec | Y | Y | 2022 | N | N | #N/A | Y | N07 | Still in review | |
| Viltepso | viltolarsen | N | N | #N/A | Y | Y | 2020 | Y | M09 | Not submitted | |
| Vitrakvi | larotrectinib sulfate | Y | N | 2019 | Y | Y | 2018 | N | L01 | ||
| Vizimpro | dacomitinib monohydrate | Y | N | 2019 | Y | Y | 2018 | N | L01 | ||
| Vonjo | pacritinib | N | N | #N/A | Y | Y | 2022 | Y | L01 | Withdrawn by the sponsor | |
| Voraxaze | glucarpidase | Y | Y | 2022 | Y | Ya | 2012 | N | V03 | ||
| Voxzogo | vosoritide | Y | Y | 2021 | Y | Y | 2021 | N | M05 | ||
| VYNDAQEL | tafamidis meglumine | Y | Ya | 2011 | Y | Y | 2019 | N | N07 | ||
| VYONDYS 53 | golodirsen | N | N | #N/A | Y | Y | 2019 | Y | M09 | Not submitted | |
| Vyvgart | efgartigimod alfa | Y | Y | 2022 | Y | Y | 2021 | N | L04 | ||
| Wakix | pitolisant | Y | Ya | 2016 | Y | Y | 2019 | N | N07 | ||
| Waylivra | volanesorsen sodium | Y | Y | 2019 | N | N | #N/A | Y | C10 | Complete Response |
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Brand name | Generic Name | Approved by EMA | EMA Approved as Orphan | EMA Approval Year | Approved by FDA | FDA Approved as Orphan | FDA Approval Year | Divergent Decision | Therapeutic Area | Reason for non-approval in EMA | Reason for non-approval in FDA |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Welireg | belzutifan | N | N | #N/A | Y | Y | 2021 | Y | L01 | Not submitted | |
| Xenpozyme | olipudase alfa | Y | Y | 2022 | Y | Y | 2022 | N | A16 | ||
| Xospata | gilteritinib fumarate | Y | Y | 2019 | Y | Y | 2018 | N | L01 | ||
| XPOVIO | selinexor | Y | N | 2021 | Y | Y | 2019 | N | L01 | ||
| Yescarta | axicabtagene ciloleucel | Y | Y | 2018 | Y | Ya | 2017 | N | L01 | ||
| Zepzelca | lurbinectedin | N | N | #N/A | Y | Y | 2020 | Y | L01 | Not submitted | |
| Zokinvy | lonafarnib | Y | Y | 2022 | Y | Y | 2020 | N | A16 | ||
| Zolgensma | onasemnogene abeparvovec | Y | Y | 2020 | Y | Y | 2019 | N | M09 | ||
| Ztalmy | ganaxolone | Y | Ya | 2023 | Y | Y | 2022 | N | N03 | ||
| Zynlonta | loncastuximab tesirine | Y | Y | 2022 | Y | Y | 2021 | N | L01 | ||
| Zynteglo | betibeglogene autotemcel | Y | Y | 2019 | Y | Y | 2022 | N | B06 |
a These data points were corrected after release of the prepublication version of the report to accurately reflect the orphan status of the drugs and decisions made by FDA and EMA.
NOTES: Ga-68-DOTATOC (gallium [68Ga] edotreotide, known as Somakit TOC in Europe) was approved as an Orphan drug by FDA in 2019 and recommended for authorization as an Orphan drug by EMA in 2016. However, FDA’s orphan designation was withdrawn or revoked on December 9, 2020. Thus, the drug did not fall into the criteria for this analysis; includes approvals outside of year range, 2018–2022, if approved by the other agency within the date range.
SOURCE: Centre for Innovation in Regulatory Science, 2024 (unpublished). Data analysis and summary to help inform the National Academies committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union. Data analysis commissioned by the committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union, National Academies of Sciences, Engineering, and Medicine, Washington, DC.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix G: List of Orphan Approvals by FDA or EMA Between 2018 and 2022." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
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