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Top 10 Worldwide Selling Orphan Drugs For 2020

October 30, 2014




EvaluatePharma has published a Report, “Orphan Drug Report 2014”, that evaluates and analyzes global orphan drug trends and developments. The following are several highlights from the Report:

•   Orphan drug sales will grow approximately 11% / year through 2020, more than double the approximately 4% / year through 2020 for drugs (excluding generics) treating larger patient populations

•   Vertex Pharmaceuticals’ combination, Kalydeco (Ivacaftor) + VX-809 (Lumacaftor), is “the world’s most valuable R&D orphan drug based on NPV (Net Present Value)”

•   Pharmacyclics’ Imbruvica (Ibrutinib) is the “most promising new orphan drug approved by the FDA in 2013”

•   For 2014, average cost/patient/year for an orphan drug is $137,782 compared to $20,875 for a non-orphan drug

•  Phase III orphan drug development cost is 50% that of non-orphans, but development time for orphan drugs is not shorter than for non-orphan drugs.

The following chart identifies the top 10 worldwide selling orphan drugs for 2020. The chart contains the following data fields:

•   Product Name/Generic Name

•   Company/ Current Status

•   FDA Indications

•   2020 Global Sales (for all indications).

Top 10 Worldwide Selling Orphan Drugs For 2020

# ProductGeneric Name * CompanyCurrent Status * FDA Indications ** 2020 Global Sales ($ Billions) *
1 Revlimid/ Lenalidomide Celgene/ Marketed Approved: Multiple Myeloma/ Mantle Cell Lymphoma/ Myelodysplastic Syndromes ODD Only: Multiple Indications $ 8.012
2 Opdivo/ Nivolumab *** Bristol-Myers Squibb/ Marketed Approved: Melanoma in Japan ODD Only: Melanoma/ Hodgkin Lymphoma $ 6.033
3 Rituxan/ Rituximab Roche/ Marketed Approved: Non-Hodgkin’sB-Cell Lymphoma/ Chronic Lymphocytic Leukemia/ WG & MPA ODD only: Immune Thrombocyto-penic Purpura $ 5.689
4 Soliris/ Eculizumab Alexion Pharmaceuticals/ Marketed Approved: aHUS/ PNH ODD Only: 6 additional unique indications $ 5.51
5 VX-809 (Lumacaftor) + Kalydeco (Ivacaftor) *** Vertex Pharmaceuticals/ Phase III Cystic Fibrosis $ 4.227
6 Keytruda/ Pembrolizumab *** Merck/ Approved Melanoma $ 3.879
7 Imbruvica/ Ibrutinib *** Pharmacyclics/ Marketed Approved: Chronic Lymphocytic Leukemia/ Mantle Cell Lymphoma ODD Only: 5 additional unique indications $ 3.602
8 Gazyva/ Obinutuzumab *** Roche/ Marketed Approved: Chronic Lymphocytic Leukemia; ODD Only: Diffuse Large B Cell Lymphoma $ 2.528
9 Obeticholic Acid Intercept Pharmaceuticals/ Phase III Primary Biliary Cirrhosis $ 2.522
10 Elelyso/ Taliglucerase Alfa Pfizer/ Marketed Gaucher Disease $ 2.48

*  Data From EvaluatePharma Report

**  Data From FDA Orphan Drug Designation (ODD) Database

*** FDA Breakthrough Therapy Designation.

Please Note: “3D Bar Graph Meeting” by “The Gold Guys Blog” [CC-BY-SA-2.0] via Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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