TheraRadar

FDA Orphan Drug Drug Approvals

233 novel drug approvals with the Orphan Drug designation, 2016–2025.

Designated for rare diseases affecting fewer than 200,000 people in the US. Sponsors receive 7 years of market exclusivity, federal tax credits for clinical research, and PDUFA fee waivers.

Key insights (2016–2025)

  • • Orphan Drug applies to 51% of all FDA novel drug approvals in this period (233 of 458 drugs).
  • • Activity has held roughly steady at ~26/year in 2023–2025 vs 2020–2022. Peak year: 2018 with 34 approvals.
  • • 147 unique sponsors received this designation; the top sponsor is Pfizer (7 approvals). Top 5 sponsors account for 15% of all Orphan Drug approvals.
  • • 78% of Orphan Drugs also received Priority Review designation — the most common pairing.

Approvals by year

2016 8 2017 18 2018 34 2019 19 2020 31 2021 25 2022 20 2023 28 2024 26 2025 24

Top sponsors

Pfizer 7 Roche 7 Bristol-Myers Squibb 7 Novartis 7 Sanofi 6 GSK 4 Bayer 4 AstraZeneca 4

All Orphan Drug approvals

2025
ANDEMBRY (GARADACIMAB-GXII) CSL Behring
2025
AVMAPKI FAKZYNJA CO-PACK (COPACKAGED) (AVUTOMETINIB POTASSIUM) VERASTEM INC
2025
BLENREP (BELANTAMAB MAFODOTIN-BLMF) GSK
2025
DAWNZERA (AUTOINJECTOR) (DONIDALORSEN SODIUM) IONIS PHARMS INC
2025
EKTERLY (SEBETRALSTAT) KALVISTA
2025
FORZINITY (ELAMIPRETIDE HYDROCHLORIDE) STEALTH BIOTHERAPS
2025
GOMEKLI (MIRDAMETINIB) SPRINGWORKS
2025
GRAFAPEX (TREOSULFAN) MEDEXUS
2025
HYRNUO (SEVABERTINIB) Bayer
2025
IBTROZI (TALETRECTINIB ADIPATE) NUVATION
2025
IMAAVY (NIPOCALIMAB-AAHU) Johnson & Johnson
2025
JASCAYD (NERANDOMILAST) Boehringer Ingelheim
2025
KOMZIFTI (ZIFTOMENIB) KURA
2025
KYGEVVI (DOXECITINE) UCB INC
2025
LYNOZYFIC (LINVOSELTAMAB-GCPT) RENGENERON PHARMACEUTICALS, INC.
2025
MODEYSO (DORDAVIPRONE HYDROCHLORIDE) CHIMERIX
2025
MYQORZO (AFICAMTEN) CYTOKINETICS
2025
PALSONIFY (PALTUSOTINE HYDROCHLORIDE) CRINETICS
2025
PENPULIMAB-KCQX AKESO BIOPHARMA
2025
QFITLIA (FITUSIRAN SODIUM) Sanofi
2025
REDEMPLO (PLOZASIRAN SODIUM) ARROWHEAD
2025
SEPHIENCE (SEPIAPTERIN) PTC THERAP
2025
WAYRILZ (RILZABRUTINIB) Sanofi
2025
YARTEMLEA (NARSOPLIMAB-WUUG) OMEROS CORP
2024
ALHEMO (CONCIZUMAB) Novo Nordisk
2024
ALYFTREK (DEUTIVACAFTOR) Vertex Pharmaceuticals
2024
AQNEURSA (LEVACETYLLEUCINE) INTRABIO
2024
ATTRUBY (ACORAMIDIS HYDROCHLORIDE) BRIDGEBIO PHARMA
2024
BIZENGRI (ZENOCUTUZUMAB-ZBCO) MERUS N.V.
2024
CRENESSITY (CRINECERFONT) NEUROCRINE
2024
DUVYZAT (GIVINOSTAT HYDROCHLORIDE) ITALFARMACO SPA
2024
HYMPAVZI (MARSTACIMAB-HNCQ) Pfizer
2024
IMDELLTRA (TARLATAMAB-DLLE) Amgen
2024
IQIRVO (ELAFIBRANOR) IPSEN
2024
LIVDELZI (SELADELPAR LYSINE) Gilead Sciences
2024
MIPLYFFA (ARIMOCLOMOL CITRATE) ZEVRA DENMARK
2024
NIKTIMVO (axatilimab-csfr) INCYTE CORP
2024
OJEMDA (TOVORAFENIB) DAY ONE BIOPHARMS
2024
PIASKY (CROVALIMAB-AKKZ) Roche
2024
REVUFORJ (REVUMENIB CITRATE) SYNDAX
2024
RYTELO (IMETELSTAT SODIUM) GERON
2024
TEVIMBRA (TISLELIZUMAB-JSGR) BEIGENE
2024
TRYNGOLZA (AUTOINJECTOR) (OLEZARSEN SODIUM) IONIS PHARMS INC
2024
VORANIGO (VORASIDENIB) SERVIER
2024
VOYDEYA (DANICOPAN) ALEXION PHARMS INC
2024
VYLOY (ZOLBETUXIMAB-CLZB) ASTELLAS
2024
WINREVAIR (SOTATERCEPT-CSRK) Merck
2024
XOLREMDI (MAVORIXAFOR) X4 PHARMS
2024
YORVIPATH (PALOPEGTERIPARATIDE) ASCENDIS PHARMA BONE
2024
ZIIHERA (ZANIDATAMAB-HRII) JAZZ PHARMS
2023
AGAMREE (VAMOROLONE) CATALYST PHARMS
2023
APHEXDA (MOTIXAFORTIDE ACETATE) AYRMID PHARMA
2023
AUGTYRO (REPOTRECTINIB) Bristol-Myers Squibb
2023
DAYBUE (TROFINETIDE) ACADIA PHARMS INC
2023
ELREXFIO (ELRANATAMAB) Pfizer
2023
FABHALTA (IPTACOPAN HYDROCHLORIDE) Novartis
2023
FILSPARI (SPARSENTAN) TRAVERE
2023
FILSUVEZ (BIRCH TRITERPENES) CHIESI
2023
JAYPIRCA (PIRTOBRUTINIB) LOXO ONCOL
2023
JOENJA (LENIOLISIB PHOSPHATE) PHARMING
2023
LAMZEDE (VELMANASE ALFA-TYCV) CHIESI FARMACEUTICI SPA
2023
LOQTORZI (TORIPALIMAB-TPZI) COHERUS BIOSCIENCES INC
2023
NGENLA (SOMATROGON-GHLA) Pfizer
2023
OGSIVEO (NIROGACESTAT HYDROBROMIDE) SPRINGWORKS
2023
OJJAARA (MOMELOTINIB DIHYDROCHLORIDE) GSK
2023
POMBILITI (CIPAGLUCOSIDASE ALFA-ATGA) AMICUS THERAP US
2023
QALSODY (TOFERSEN) Biogen
2023
REZZAYO (REZAFUNGIN ACETATE) MUNDIPHARMA
2023
RIVFLOZA (NEDOSIRAN SODIUM) Novo Nordisk
2023
RYSTIGGO (ROZANOLIXIZUMAB-NOLI) UCB INC
2023
SKYCLARYS (OMAVELOXOLONE) Biogen
2023
SOHONOS (PALOVAROTENE) IPSEN
2023
TALVEY (TALQUETAMAB-TGVS) Johnson & Johnson
2023
VANFLYTA (QUIZARTINIB DIHYDROCHLORIDE) DAIICHI SANKYO INC
2023
VEOPOZ (POZELIMAB-BBFG) Regeneron
2023
WAINUA (AUTOINJECTOR) (EPLONTERSEN SODIUM) AstraZeneca
2023
ZILBRYSQ (ZILUCOPLAN SODIUM) UCB INC
2023
ZYNYZ (RETIFANLIMAB-DLWR) INCYTE CORP
2022
AMVUTTRA (VUTRISIRAN SODIUM) ALNYLAM PHARMS INC
2022
BRIUMVI (UBLITUXIMAB-XIIY) TG THERAPEUTICS, INC
2022
CAMZYOS (MAVACAMTEN) Bristol-Myers Squibb
2022
ELAHERE (MIRVETUXIMAB SORAVTANSINE-GYNX) IMMUNOGEN INC
2022
ENJAYMO (SUTIMLIMAB-JOME) RECORDATI RARE DISEASES, INC.
2022
IMJUDO (TREMELIMUMAB-ACTL) AstraZeneca
2022
KIMMTRAK (TEBENTAFUSP-TEBN) IMMUNOCORE LTD
2022
KRAZATI (ADAGRASIB) Bristol-Myers Squibb
2022
LUNSUMIO (MOSUNETUZUMAB-AXGB) Roche
2022
LYTGOBI (FUTIBATINIB) TAIHO ONCOLOGY
2022
OPDUALAG (NIVOLUMAB) Bristol-Myers Squibb
2022
PLUVICTO (LUTETIUM LU-177 VIPIVOTIDE TETRAXETAN) Novartis
2022
PYRUKYND (MITAPIVAT SULFATE) AGIOS PHARMS INC
2022
RELYVRIO (SODIUM PHENYLBUTYRATE) AMYLYX
2022
SPEVIGO (SPESOLIMAB-SBZO) Boehringer Ingelheim
2022
SUNLENCA (LENACAPAVIR SODIUM) Gilead Sciences
2022
TECVAYLI (TECLISTAMAB-CQYV) Johnson & Johnson
2022
TERLIVAZ (TERLIPRESSIN ACETATE) MALLINCKRODT IRELAND
2022
VONJO (PACRITINIB CITRATE) SOBI
2022
XENPOZYME (OLIPUDASE ALFA-RPCP) Sanofi
2021
AMONDYS 45 (CASIMERSEN) Sarepta Therapeutics
2021
BESREMI (ROPEGINTERFERON ALFA-2B-NJFT) PHARMAESSENTIA CORP
2021
BYLVAY (ODEVIXIBAT) IPSEN
2021
CYTALUX (PAFOLACIANINE SODIUM) ON TARGET LABS
2021
EMPAVELI (PEGCETACOPLAN) APELLIS PHARMS
2021
EVKEEZA (EVINACUMAB-DGNB) Regeneron
2021
EXKIVITY (MOBOCERTINIB SUCCINATE) Takeda
2021
FEXINIDAZOLE Sanofi
2021
LIVMARLI (MARALIXIBAT CHLORIDE) MIRUM
2021
LIVTENCITY (MARIBAVIR) Takeda
2021
LUMAKRAS (SOTORASIB) Amgen
2021
NEXVIAZYME (AVALGLUCOSIDASE ALFA-NGPT) Sanofi
2021
NULIBRY (FOSDENOPTERIN HYDROBROMIDE) SENTYNL THERAPS INC
2021
REZUROCK (BELUMOSUDIL MESYLATE) KADMON PHARMS LLC
2021
RYLAZE (ASPARAGINASE ERWINIA CHRYSANTHEMI (RECOMBINANT)-RYWN) JAZZ PHARMS
2021
SCEMBLIX (ASCIMINIB HYDROCHLORIDE) Novartis
2021
SKYTROFA (LONAPEGSOMATROPIN-TCGD) ASCENDIS PHARMA ENCOCRINOLOGY DIV A/S
2021
TAVNEOS (AVACOPAN) CHEMOCENTRYX
2021
TEPMETKO (TEPOTINIB HYDROCHLORIDE) EMD SERONO INC
2021
TRUSELTIQ (INFIGRATINIB PHOSPHATE) HELSINN HLTHCARE
2021
UKONIQ (UMBRALISIB TOSYLATE) TG THERAPS
2021
VOXZOGO (VOSORITIDE) BIOMARIN PHARM
2021
VYVGART (EFGARTIGIMOD ALFA) ARGENX BV
2021
WELIREG (BELZUTIFAN) Merck
2021
ZYNLONTA (LONCASTUXIMAB TESIRINE-LPYL) ADC Therapeutics SA
2020
ARTESUNATE AMIVAS
2020
AYVAKIT (AVAPRITINIB) BLUEPRINT MEDICINES
2020
DANYELZA (NAXITAMAB-GQGK) Y-MABS THERAPEUTICS INC
2020
DETECTNET (COPPER CU-64 DOTATATE) CURIUM
2020
DOJOLVI (TRIHEPTANOIN) ULTRAGENYX PHARM INC
2020
EBANGA (ANSUVIMAB-ZYKL) RIDGEBACK BIOTHERAPEUTICS
2020
ENSPRYNG (SATRALIZUMAB) Roche
2020
EVRYSDI (RISDIPLAM) Roche
2020
GAVRETO (PRALSETINIB) BLUEPRINT MEDICINES
2020
IMCIVREE (SETMELANOTIDE ACETATE) RHYTHM
2020
INMAZEB (ATOLTIVIMAB) Regeneron
2020
INQOVI (CEDAZURIDINE) TAIHO ONCOLOGY
2020
ISTURISA (OSILODROSTAT PHOSPHATE) RECORDATI RARE
2020
KOSELUGO (SELUMETINIB SULFATE) AstraZeneca
2020
LAMPIT (NIFURTIMOX) Bayer
2020
MONJUVI (TAFASITAMAB-CXIX) MORPHOSYS US INC
2020
ORLADEYO (BEROTRALSTAT DIHYDROCHLORIDE) BIOCRYST
2020
OXLUMO (LUMASIRAN SODIUM) ALNYLAM PHARMS INC
2020
PEMAZYRE (PEMIGATINIB) INCYTE CORP
2020
QINLOCK (RIPRETINIB) DECIPHERA PHARMS
2020
RETEVMO (SELPERCATINIB) Eli Lilly
2020
SARCLISA (ISATUXIMAB-IRFC) Sanofi
2020
TABRECTA (CAPMATINIB HYDROCHLORIDE) Novartis
2020
TAZVERIK (TAZEMETOSTAT HYDROBROMIDE) EPIZYME INC
2020
TEPEZZA (TEPROTUMUMAB-TRBW) HORIZON THERAPEUTICS IRELAND
2020
TRODELVY (SACITUZUMAB GOVITECAN-HZIY) IMMUNOMEDICS INC
2020
TUKYSA (TUCATINIB) SEAGEN
2020
UPLIZNA (INEBILIZUMAB-CDON) VIELA BIO
2020
VILTEPSO (VILTOLARSEN) NIPPON SHINYAKU
2020
ZEPZELCA (LURBINECTEDIN) JAZZ
2020
ZOKINVY (LONAFARNIB) SENTYNL THERAPS INC
2019
ADAKVEO (CRIZANLIZUMAB-TMCA) Novartis
2019
BRUKINSA (ZANUBRUTINIB) BEONE MEDICINES USA
2019
CABLIVI (CAPLACIZUMAB-YHDP) ABLYNX NV
2019
EGATEN (TRICLABENDAZOLE) Novartis
2019
GIVLAARI (GIVOSIRAN SODIUM) ALNYLAM PHARMS INC
2019
INREBIC (FEDRATINIB HYDROCHLORIDE) Bristol-Myers Squibb
2019
OXBRYTA (VOXELOTOR) GLOBAL BLOOD THERAPS
2019
POLIVY (POLATUZUMAB VEDOTIN-PIIQ) Roche
2019
PRETOMANID Viatris
2019
REBLOZYL (LUSPATERCEPT-AAMT) Bristol-Myers Squibb
2019
ROZLYTREK (ENTRECTINIB) Roche
2019
SCENESSE (AFAMELANOTIDE) CLIVUNEL INC
2019
SUNOSI (SOLRIAMFETOL HYDROCHLORIDE) AXSOME MALTA
2019
TRIKAFTA (COPACKAGED) (ELEXACAFTOR, IVACAFTOR, TEZACAFTOR) Vertex Pharmaceuticals
2019
TURALIO (PEXIDARTINIB HYDROCHLORIDE) DAIICHI SANKYO INC
2019
VYNDAQEL (TAFAMIDIS MEGLUMINE) FOLDRX PHARMS
2019
VYONDYS 53 (GOLODIRSEN) Sarepta Therapeutics
2019
WAKIX (PITOLISANT HYDROCHLORIDE) HARMONY
2019
XPOVIO (SELINEXOR) KARYOPHARM THERAPS
2018
ASPARLAS (CALASPARGASE PEGOL-MKNL) SERVIER PHARMA LLC
2018
BRAFTOVI (ENCORAFENIB) ARRAY BIOPHARMA INC
2018
COPIKTRA (DUVELISIB) SECURA
2018
CRYSVITA (BUROSUMAB-TWZA) KYOWA KIRIN
2018
DAURISMO (GLASDEGIB MALEATE) Pfizer
2018
DIACOMIT (STIRIPENTOL) BIOCODEX SA
2018
ELZONRIS (TAGRAXOFUSP-ERZS) STEMLINE THERAPEUTICS INC
2018
EPIDIOLEX (CANNABIDIOL) JAZZ PHARMS RES
2018
FIRDAPSE (AMIFAMPRIDINE PHOSPHATE) CATALYST PHARMS
2018
GALAFOLD (MIGALASTAT HYDROCHLORIDE) AMICUS THERAP US
2018
GAMIFANT (EMAPALUMAB-LZSG) NOVIMMUNE S.A.
2018
KRINTAFEL (TAFENOQUINE SUCCINATE) GSK
2018
LORBRENA (LORLATINIB) Pfizer
2018
LUMOXITI (MOXETUMOMAB PASUDOTOX-TDFK) INNATE PHARMA
2018
LUTATHERA (LUTETIUM LU 177 DOTATATE) AAA USA INC
2018
MEKTOVI (BINIMETINIB) ARRAY BIOPHARMA INC
2018
MOXIDECTIN MDGH
2018
OMEGAVEN (FISH OIL TRIGLYCERIDES) Fresenius Kabi
2018
ONPATTRO (PATISIRAN SODIUM) ALNYLAM PHARMS INC
2018
OXERVATE (CENEGERMIN-BKBJ) DOMPE FARMACEUTICI
2018
PALYNZIQ (PEGVALIASE-PQPZ) BIOMARIN PHARM
2018
POTELIGEO (MOGAMULIZUMAB-KPKC) KYOWA KIRIN
2018
REVCOVI (ELAPEGADEMASE-LVLR) CHIESI USA INC
2018
SYMDEKO (COPACKAGED) (IVACAFTOR) Vertex Pharmaceuticals
2018
TAKHZYRO (LANADELUMAB-FLYO) DYAX CORP.
2018
TAVALISSE (FOSTAMATINIB DISODIUM) RIGEL PHARMS
2018
TEGSEDI (INOTERSEN SODIUM) AKCEA THERAPS
2018
TIBSOVO (IVOSIDENIB) SERVIER
2018
TPOXX (TECOVIRIMAT) SIGA TECHNOLOGIES
2018
TROGARZO (IBALIZUMAB-UIYK) THERATECHNOLOGIES
2018
ULTOMIRIS (RAVULIZUMAB-CWVZ) ALEXION PHARM
2018
VITRAKVI (LAROTRECTINIB SULFATE) Bayer
2018
VIZIMPRO (DACOMITINIB) Pfizer
2018
XOSPATA (GILTERITINIB FUMARATE) ASTELLAS
2017
ALIQOPA (COPANLISIB DIHYDROCHLORIDE) Bayer
2017
ALUNBRIG (BRIGATINIB) Takeda
2017
AUSTEDO (DEUTETRABENAZINE) Teva
2017
BAVENCIO (AVELUMAB) EMD SERONO INC
2017
BENZNIDAZOLE CHEMO RESEARCH SL
2017
BESPONSA (INOTUZUMAB OZOGAMICIN) Pfizer
2017
BRINEURA (CERLIPONASE ALFA) BIOMARIN PHARM
2017
CALQUENCE (ACALABRUTINIB MALEATE) AstraZeneca
2017
EMFLAZA (DEFLAZACORT) PTC THERAP
2017
HEMLIBRA (EMICIZUMAB) Roche
2017
IDHIFA (ENASIDENIB MESYLATE) Bristol-Myers Squibb
2017
MACRILEN (MACIMORELIN ACETATE) AETERNA ZENTARIS
2017
MEPSEVII (VESTRONIDASE ALFA-VJBK) ULTRAGENYX PHARM INC
2017
PREVYMIS (LETERMOVIR) Merck
2017
RADICAVA (EDARAVONE) SHIONOGI
2017
RYDAPT (MIDOSTAURIN) Novartis
2017
XERMELO (TELOTRISTAT ETIPRATE) TERSERA
2017
ZEJULA (NIRAPARIB TOSYLATE) GSK
2016
DEFITELIO (DEFIBROTIDE SODIUM) JAZZ PHARMS
2016
EXONDYS 51 (ETEPLIRSEN) Sarepta Therapeutics
2016
LARTRUVO (OLARATUMAB) Eli Lilly
2016
NUPLAZID (PIMAVANSERIN TARTRATE) ACADIA PHARMS INC
2016
OCALIVA (OBETICHOLIC ACID) INTERCEPT PHARMS INC
2016
RUBRACA (RUCAPARIB CAMSYLATE) PHARMAAND
2016
SPINRAZA (NUSINERSEN SODIUM) Biogen
2016
VENCLEXTA (VENETOCLAX) AbbVie

Other FDA designations

First-in-Class Breakthrough Therapy Accelerated Approval Priority Review Fast Track

Designation data from FDA Center for Drug Evaluation and Research (CDER) annual New Drug Therapy Approvals reports, 2016–2025. Each drug is counted once per the year of its novel approval; many drugs hold multiple designations.

Browse all FDA approvals → | Novel approvals dashboard →