Orphan Drug Trends: A data-driven analysis
The commercial and market access benefits that arise from gaining an orphan drug designation have ensured continued interest from pharma in this approach.
So, what does the recent landscape for orphan drugs with rare disease approval look like?
Which companies are leading the way?
What therapy areas are they targeting?
Rich in charts, tables and data, this report and data set provides a detailed overview of 200 orphan drugs approved in the US for a rare disease indication since 2019.
- Subject synopsis
- Research methodology and objectives
- Overview
- Orphan drugs in the US 2003-2023
- US orphan drugs by therapy area 2003-2023
- Newly approved orphan drugs 2019-2023
- Therapy area analysis
- Anti-infectives overview
- Recent anti-infective orphan drug approvals
- Descovy
- Dificid
- Ebanga
- Epclusa
- Harvoni
- Inmazeb
- Livtencity
- Mavyret
- Noxafil
- Pretomanid
- Prevymis
- Rebyota
- Rezzayo
- Sovaldi
- Tembexa
- Vowst
- Dermatology overview
- Recent dermatology orphan drug approvals
- Actemra
- NexoBrid
- Octagam 10%
- Ofev
- Spevigo
- Stratagraft
- Vyjuvek
- Genitourinary overview
- Recent genitourinary orphan drug approvals
- Filspari
- Oxlumo
- Tarpeyo
- Terlivaz
- Ultomiris
- Haematology overview
- Recent haematology orphan drug approvals
- Adakveo
- Altuviiio
- Besremi
- Empaveli
- Enjaymo
- Hemgenix
- Orladeyo
- Oxbryta
- Pyrukynd
- Rebinyn
- Reblozyl
- Takhzyro
- Ultomiris
- Vonjo
- Vonvendi
- Zynteglo
- Immunology overview
- Recent immunology orphan drug approvals
- Biktarvy
- Dupixent
- Enspryng
- Joenja
- Omisirge
- Soliris
- Tybost
- Uplizna
- Inflammatory disorders overview
- Recent anti-inflammatory orphan drug approvals
- Humira
- Ilaris
- Kineret
- Otezla
- Ryplazim
- Simponi Aria
- Welireg
- Metabolic disorders overview
- Recent metabolic orphan drug approvals
- Cuvrior
- Dojolvi
- Givlaari
- Imcivree
- Isturisa
- Lamzede
- Livmarli
- Nexviazyme
- Pheburane
- Procysbi
- Recorlev
- Zokinvy
- Musculoskeletal disorders overview
- Recent musculoskeletal orphan drug approvals
- Amondys 45
- Crysvita
- Emflaza
- Evrysdi
- Mycapssa
- Ultomiris
- Viltepso
- Voxzogo
- Vyondys 53
- Vyvgart
- Zolgensma
- Neuroscience overview
- Recent neuroscience orphan drug approvals
- Daybue
- Diacomit
- Epidiolex
- Exservan
- Fintepla
- Firdapse
- Hetlioz
- Hyftor
- Imcivree
- Koselugo
- Lumryz
- Nayzilam
- Nulibry
- Qalsody
- Radicava ORS
- Relyvrio
- Sezaby
- Skyclarys
- Skysona
- Sunosi
- Tiglutik
- Valtoco
- Wakix
- Xywav
- Ztalmy
- Oncology overview
- Recent oncology orphan drug approvals
- Abecma
- Adcetris
- Alunbrig
- Ayvakit
- Breyanzi
- Brukinsa
- Cabometyx/Cometriq
- Calquence
- Carvykti
- Cotellic
- Cyramza
- Danyelza
- Darzalex
- Elahere
- Enhertu
- Exkivity
- Fyarro
- Gavreto
- Imfinzi
- Imjudo
- Inqovi
- Inrebic
- Jaypirca
- Jelmyto
- Keytruda
- Kimmtrak
- Krazati
- Kymriah
- Lumakras
- Lunsumio
- Lynparza
- Lytgobi
- Mekinist
- Monjuvi
- Onureg
- Opdivo
- Opdualag
- Pemazyre
- Polivy
- Pomalyst
- Rezlidhia
- Retevmo
- Rezurock
- Rituxan
- Rozlytrek
- Rylaze
- Sarclisa
- Scemblix
- Tabrecta
- Tafinlar
- Tagrisso
- Tazverik
- Tecartus
- Tecentriq
- Tecvayli
- Tepmetko
- Tibsovo
- Tukysa
- Turalio
- Vidaza
- Xalkori
- Xpovio
- Yervoy
- Yescarta
- Zepzelca
- Zynlonta
- Zynyz
- Ophthalmology overview
- Recent ophthalmic orphan drug approvals
- Eylea
- Tepezza
- Verkazia
- Respiratory overview
- Recent respiratory orphan drug approvals
- Bronchitol
- Kalydeco
- Orkambi
- Symdeko
- Trikafta
- Systemic/multisystem disorders overview
- Recent systemic/multisystem disorder orphan drug approvals
- Amvuttra
- Ayvakit
- Darzalex Faspro
- Scenesse
- Anti-infectives overview
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