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Orphan Drugs And Rare Diseases: 2014 FDA CDER Expedited Program Statistics

January 21, 2015

In January 2015, FDA’s CDER (Center for Drug Evaluation and Research) publishes its fourth annual report, “Novel New Drugs Summary”, for calendar year 2014. The report identifies the approved 41 novel new drugs, approved as New Molecular Entities (NMEs) under New Drug Applications (NDAs) or as new therapeutic biologics under Biologics License Applications (BLAs).

Following are several highlights of the Report on orphan drugs, rare diseases, and the FDA Breakthrough Therapy Designation:

•    For 2005 – 2013, FDA’s CDER averaged approximately 25 novel new drug approvals per year

•    For 2014, FDA’s CDER approves 41 novel new drugs

•    41.5 % (17 of 41) of the novel new drugs approved in 2014 are for treatment of a rare or “orphan disease” – more than any previous year.

CDER uses 4 expedited development and review pathways to expedite or speed up the approval of novel drugs:

•   Fast Track

•   Breakthrough Therapy Designation

•   Priority Review

•   Accelerated Approval.

The chart below presents data from the “Novel New Drugs 2014 Summary” Report for the 17 CDER approved novel drugs for 2014 along with identifying what 4 of CDER’s expedited development and review pathways are used for each orphan drug.

From this chart below, the following can be observed for the 17 novel orphan drugs CDER approved for 2014:

•     59 % of approved orphan drugs receive Fast Track status

•     41 % of approved orphan drugs receive Breakthrough Therapy Designation

•   100 % of approved orphan drugs receive Priority Review status

•     47 % of approved orphan drugs receive Accelerated Approval.

17 2014 FDA CDER New Molecular Entities/Therapeutic Biological Approved Orphan Products 

Drug Name/ Generic Name Orphan Designation Fast Track Break-Through Priority Review Accelerated Approval
Beleodaq/ Belinostat X X X X
Blincyto/ Blinatumomab X X X X
Cerdelga/ Eliglustat X X
Cyramza/ Ramucirumab X X X
Esbriet/ Pirfenidone X X X X
Hetlioz/ Tasimelteon X X
Impavido/ Miltefosine X X X
Keytruda/ Pembrolizumab X X X X
Lynparza/ Olaparib X X X
Myalept/ Metreleptin for injection X X X
Northera/ Droxidopa X X X X
Ofev/ Nintedanib X X X X
Opdivo/ Nivolumab X X X X X
Sylvant/ Siltuximab X X
Vimizim/ Elosulfase Alfa X X X
Zydelig/ Idelalisib X X X X X
Zykadia/ Ceritinib X X X X

Source of Data: FDA’s CDER “Novel New Drugs 2014 Summary” Report

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

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