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The Orphan Drug Machine

Interactive: How Orphan Drugs Win The ‘Monopoly’ Game

Check out all the drugs the FDA has approved to treat rare diseases. You can search by brand name, or by disease, and see familiar names that were first sold on the mass market or all the drugs that won FDA approval to treat more than one rare disease. Each approval gives the drugmaker seven years of exclusive rights to the market. Drugmakers are companies that sought orphan approval, not necessarily current drug owners. Some drugs appear in more than one tab.

Search for drug or disease
Anavip Anthim Apokyn Arcalyst
Raxibacumab/Abthraxtm Acetadote Acthrel Adagen
Adcirca Agrylin Aldurazyme Alecensa
Alimta Alkeran For Injection Aloprim Alphanine
Alprolix Proamatine/Amatine Ammonul Ampyra
Anascorp Aromasin Arranon Atnativ
Atryn Avonex Babybig Banzel
Beleodaq Bendeka Benefix Berinert
Betapace Betaseron Bexxar Blincyto
Bosulif Buphenyl Busulfex Cafcit
Campath Caprelsa Carbaglu Cayston
Ceprotin Cerdelga Cerebyx Ceredase
Cerezyme Cetylev Chemet Chenix
Chirhostim Cholbam Cibacalcin Cinryze
Clolar Cnj-016 Coagadex Coartem
Colcrys Cometriq Copaxone Crofab
Curosurf Cuvposa Cyanokit Cystadane
Cystagon Cystaran Dacogen Daunoxome
Defitelio Depocyt Digibind Duopa
Duraclon Elaprase Eldepryl Elelyso
Elitek Ellence Elliotts B Solution Elmiron
Eloctate Envarsus Xr Erwinaze Esbriet
Ethamolin Evomela Exosurf Neonatal Fabrazyme
Fareston Farydak Felbatol Ferriprox
Firazyr Fludara Folotyn Galzin
Gamimune N Gastrocrom Gattex Gralise
Halfan Hemangeol Hetlioz Hexalen
Hivid Humate-P Idamycin Idelvion
Ifex Imlygic Impavido Increlex
Infasurf Inomax Iplex Juxtapid
Kalydeco Kanuma Kogenate Korlym
Krystexxa Kuvan Kynamro Kyprolis
Lamprene Lenvima Letairis Leustatin
Lidoderm Lioresal Lutrepulse Luveris
Lynparza Makena Marqibo Mekinist
Membraneblue Mesnex Metrodin Metrogel
Mitosol Moctanin Mononine Mozobil
Myalept Mycobutin Myobloc Anthrasil
Botulism antitoxin heptavalent (A\, B\, C\, D\, E\, F\, G) (Equine) Cotellic Spherusol Stimate
Diastat Acudial Pentetate Calcium Trisodium Oforta Praxbind
Onivyde Portrazza Phoxillum Qualaquin
Vonvendi Xuriden Naglazyme Natpara
Nebupent Neoprofen Netspot Neumega
Neutrexin Ninlaro Nipent Nithiodote
Normocarb Hf Northera Nplate Nulojix
Nutrestore Nymalize Obizur Ocaliva
Ofev Oncaspar Oncoscint Cr/Ov Onfi
Ontak Opsumit Opticrom Orlaam
Ornidyl Panhematin Panretin Parathar
Paser Pentam Pentaspan
Pomalyst Priftin Prolastin-C Protropin
Provayblue Provigil Pulmozyme Purixan
Qutenza Radiogardase Ravicti Refacto\, Xyntha
Refludan Remodulin Renacidin Repatha
Respigam Retisert Retrovir Riastap
Rifadin Rifater Rilutek Rixubis
Ruconest Ryanodex Sabril Saizen
Sclerosol Serostim Simulect Sirturo
So-Aqueous Somavert Stivarga Strensiq
Suboxone Subutex Sucraid Sulfamylon
Supprelin Supprelin La Sylvant Synribo
Syprine Tafinlar Tagrisso Targretin
Thiola Thrombate Iii Tobi Torisel
Totect Tracleer Trasylol Tretten
Triostat Trisenox Tyvaso Ucephan
Unituxin Uptravi Urso 250 Valchlor
Valstar Varizig Venclexta Ventavis
Vesanoid Vidaza Vimizim Vitrasert
Voraxaze Vpriv Vumon Wilate
Winrho Sd Xalkori Xenazine Yondelis
Zavesca Zelboraf Zenapax Zevalin
Zinecard Zirgan Zolinza Zometa\, Zabel
Zorbtive Zykadia Carnexiv Empliciti
Exondys 51 Vermox Nutropin Depot Tasigna
Thyroshield Extraneal (With 7.5% Icodextrin) Peritoneal Dialysis Solutio Mylotarg Alomide Ophthalmic Solution

Approved Orphan Uses


    We relied primarily on the FDA’s orphan drug designations and approvals database because it has the most detailed information about orphan drug approvals, disease indications and dates. We included only orphan designations that led to approvals, and cleaned up and standardized many of the drug names manually because they had either changed or had never been entered.

    We also used the orphan drugs database to look for drugs approved for multiple orphan indications. Occasionally, a drug had more than one approval on the same day. Sometimes, they were months apart, and other times they were years apart.

    Determining whether a drug was on the mass market before its orphan use was approved required pulling initial drug approval dates from additional FDA datasets, including the Drugs@FDA database as well as lists of biologic drugs and fractionated plasma products that didn’t appear in Drugs@FDA. If a drug had an initial approval date before its first marketing approval date in the orphan database, we put it in the mass market first category.

    Our analysis does not include a list of drugs that were first approved for orphan indications and later received approval for mass market indications. Although the FDA tried to help us with this, we determined that data was not consistent enough for our project.

    Our analysis does not examine the medical or cost effectiveness of these drugs.

    Sydney Lupkin and Elizabeth Lucas

    Lookup tool built by Alley Interactive; Embed code is available for organizations who want to publish. Contact for more information.

    KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.

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