. Author manuscript; available in PMC: 2013 Oct 1.

Published in final edited form as: Ann Neurol. 2012 Oct;72(4):481–490. doi: 10.1002/ana.23672

Table 2.

Blockbuster drugs that have obtained orphan drug designation²⁷. The highlighting drugs are primarily designated or frequently used for neurological conditions.

Brand Name	Generic Name	Main Indications	Designation (Year)	Global Sales^a 2010 (Million)
Humira	Adalimumab	Inflammatory bowel diseases, Juvenile rheumatoid arthritis	2005^*	6,548
Fosamax ^b	Alendronate	Osteogenesis Imperfecta	2001	-
Ceredase^#	Alglucerase	Type I, II, III Gaucher’s disease	1985^*	720
Abilify	Aripiprazole	Tourette’s syndrome	2006	2,565
Avastin	Bevacizumab	Chemotherapy for cancers	2003	7,178
Velcade	Bortezomib	Multiple myeloma, lymphomas	2003^*	-
Tracleer	Bosentan	Pulmonary artery hypertension, Idiopathic pulmonary fibrosis	2000^*	-
Botox	Botulinum toxin	Cerebral palsy, Dystonia	1984^*	1,414
Novoseven	Factor VIIa	Hemophilia, Factor VII deficiency, Intracranial hemorrphage	1988^*	1,483
Epogen	Epoetin alfa	Anemia related to HIV infection,, Anemia in end stage renal disease,	1986^*	2,524
Procrit	Epoetin alfa	Anemia related to HIV infection,, Anemia in end stage renal disease,	1987	1,934
Enbrel	Etanercept	Juvenile rheumatoid arthritis, Wegener’s granulomatosis	1998^*	7,287
Neupogen	Filgrastim	Neutropenia in various conditions	1990^*	1,286
Neurontin ^b	Gabapentin	Treatment of ALS	1995	823
Copaxone	Glatiramer	Multiple sclerosis	1987^*	3,300
Gleevec	Imatinib	Acute and chronic leukemia	2001^*	4,265
Cerezyme^#	Imiglucerase	Type I, II, III Gaucher’s disease	1991^*	720
Remicade	Infliximab	Inflammatory bowel diseases,	1995^*	6,565
Betaseron	IFN beta-1b	Multiple sclerosis	1988^*	1,658
Avonex	IFN beta-1a	Multiple sclerosis	1991^*	2,518
Rebif	IFN beta-1a	Multiple sclerosis	1992	2,297
Lamictal ^b	Lamotrigine	Lennox-Gastaut Sundrome	1995^*	671
Revlimid	Lenalidomide	Multiple myeloma, Lymphoma, Leukemia	2001^*	-
Lupron	Leuprolide	Precocious puberty	1988^*	1,617
Mobic	Meloxicam	Juvenile rheumatoid arthritis	2002^*
Provigil ^b	Modafinil	Narcolepsy	1993^*	999
CellCept	Mycophenolate	Pemphigus vulgaris, MG	2006	1,433
Sandostatin	Octreotide	Acromegaly	1998^*	1,291
Kogenate	Octocog	Hemophilia	1989^*	1,380
Taxol	Paclitaxel	Kaposi’s sarcoma	1997^*	-
Pegasys	IFN alfa-2^a	Renal cell carcinoma, CML	1998	1,828
Alimta	Pemetrexed	Malignant mesothelioma	2001^*	-
Mirapex ^b	Pramipexole	Tourette’s syndrome	2008	-
Evista	Raloxifene	Postmenopausal breast cancer	2005^*	-
Rituxan	Rituximab	NHL, chronic leukemia, vasculitis	1994^*	7,061
Vioxx	Rofecoxib	Juvenile rheumatoid arthritis	2004	-
Prograf	Tacrolimus	Graft-versus-host-disease	1998^*	-
Cialis	Tadalafil	Pulmonary artery hypertension	2006	1,699
Temodar	Temozolomide	Malignant glioma, Metastatin melanoma	1998^*	1,065
Spiriva	Tiotropium	Cystic fibrosis (conjuctive therapy)	2008	3,935
Topamax ^b	Topiramate	Lennox-Gastaut Sundrome	1992^*	-
Herceptin	Trastuzumab	Pancreatic cancer	1999	6,032
Zometa	Zoledronic acid	Tumor induced hypercalcemia	2000^*	1,511

Sources: FDA, Pipeline Review, Drug Topics, and Pharmacy Times

Numbers represent million dollars

Generic formulation is now available

Received orphan approval