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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
Acetadote Acthrel Adagen Adcirca
Adreview Agrylin Aldurazyme Alecensa
Alimta Alinia Alkeran For Injection Alomide Ophthalmic Solution
Aloprim Alphanine Alprolix Ammonul
Ampyra Anascorp Anavip Anthim
Anthrasil Apokyn Arcalyst Aromasin
Arranon Atnativ Atryn Avonex
Babybig Banzel Beleodaq Bendeka
Benefix Berinert Betapace Betaseron
Bexxar Blincyto Bosulif Botulism antitoxin heptavalent (A, B, C, D, E, F, G) (Equine)
Buphenyl Busulfex Cafcit Campath
Caprelsa Carbaglu Carnexiv Cayston
Ceprotin Cerdelga Cerebyx Ceredase
Cerezyme Cetylev Chemet Chenix
Chirhostim Cholbam Cibacalcin Cinryze
Clolar Cnj-016 Coagadex Coartem
Colcrys Cometriq Copaxone Cotellic
Crofab Curosurf Cuvposa Cyanokit
Cystadane Cystagon Cystaran Dacogen
Daunoxome Defitelio Depocyt Diastat Acudial
Digibind Duopa Duraclon Elaprase
Eldepryl Elelyso Elitek Ellence
Elliotts B Solution Elmiron Eloctate Empliciti
Envarsus Xr Erwinaze Esbriet Ethamolin
Evomela Exondys 51 Exosurf Neonatal Extraneal
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
Membraneblue Mesnex Metrodin Metrogel
Mitosol Moctanin Mononine Mozobil
Myalept Mycobutin Mylotarg Myobloc
Naglazyme Natpara Nebupent Neoprofen
Netspot Neumega Neutrexin Ninlaro
Nipent Nithiodote Normocarb Hf Northera
Nplate Nulojix Nutrestore Nutropin Depot
Nymalize Obizur Ocaliva Ofev
Oforta Oncaspar Oncoscint Cr/Ov Onfi
Onivyde Ontak Opsumit Opticrom
Orlaam Ornidyl Panhematin Panretin
Parathar Paser Pentam Pentaspan
Pentetate Calcium Trisodium Phoslo Phoxillum Pomalyst
Portrazza Praxbind Priftin Proamatine/Amatine
Prolastin-C Protropin Provayblue Provigil
Pulmozyme Purixan Qualaquin Qutenza
Radiogardase Ravicti Raxibacumab/Abthraxtm Refacto, Xyntha
Refludan Remodulin Renacidin Repatha
Respigam Retisert Retrovir Riastap
Rifadin Rifater Rilutek Rixubis
Ruconest Rubraca Ryanodex Sabril
Saizen Sclerosol Serostim Simulect
Sirturo So-Aqueous Somavert Spherusol
Spinraza Stimate Stivarga Strensiq
Suboxone Subutex Sucraid Sulfamylon
Supprelin Supprelin La Sylvant Synribo
Syprine Tagrisso Targretin Tasigna
Thiola Thrombate III Thyroshield Tobi
Torisel Totect Tracleer Trasylol
Tretten Triostat Trisenox Tyvaso
Ucephan Unituxin Uptravi Urso 250
Vaccinia Immune Globulin (Human) Intravenous Valchlor Valstar Varizig
Venclexta Ventavis Vermox Vesanoid
Vidaza Vimizim Vitrasert Vonvendi
Voraxaze Vpriv Vumon Wilate
Winrho Sd Xalkori Xenazine Yondelis
Zavesca Zelboraf Zenapax Zevalin
Zinecard Zirgan Zolinza Zometa, Zabel
Zorbtive Zykadia

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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