Tuesday, December 16, 2014

37% of New Drugs Approved by FDA in 2014 were for Treatment of Rare Diseases

New data from the FDA (here) shows that 2014 was a banner year for orphan drugs, which are drugs that treat "rare" diseases/disorders affecting fewer than 200,000 people in the U.S.

The drug approval data from 1994 through 2014 (to date) are plotted in the following charts:

NME=New Molecular Entity, BLA=Biologic License Application; Source: FDA

Let's dig deeper into the numbers.

A total of 41 new molecular entities (NMEs) and biologics (BLAs) were approved by the FDA in 2014 - 15 of those were approved in December alone (11 in final two weeks!). That's the highest number of approvals since 1996.

Orphan drug approvals represent 37% (15 out 41) of all new drug approvals in 2014. Today, orphan drugs have the potential to turn into blockbusters with annual sales over $1 Billion (read, for example, "New Big Pharma Economies of Scale: Less Patients Needed to Reach Blockbuster Sales").

2014 was also a very good year for biologics. Ten (10) new licenses to market biologic drugs were issued to date in 2014. That's more than any other year and represents 24% of all new drug approvals.

[This post was updated on 2-JAN-2015 with new data.]

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