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A Bright Future for Orphan Disease Indications

March 5, 2013
In celebration of National Rare Disease Day BIO’s Industry Analysis team took a look at recently-approved drugs in the BioMedTracker database with an orphan indication designation. Our research found that 2012 saw the greatest number of approvals of therapies for orphan indications going back all the way to 2000 (This count includes previously approved drugs with new, orphan indication approvals).

 Historic Orphan Drug Approvals

Overall, 24 therapies were approved in orphan indications last year, including drugs for Anthrax, Gaucher’s Disease, Multiple Myeloma, and Cystic Fibrosis.  The buzz from BIO’s recent BIO CEO & Investor Conference confirms that the orphan drug space is also at the top of investors’ minds: BIO’s 2013 survey of buy-side investors showed that companies in the orphan/rare disease space doubled in popularity over 2012 as investors weighed investment opportunities across therapeutic areas.

We also looked into BioMedTracker’s orphan designation pipeline and found 18 treatments in the NDA/BLA filing phase, 215 in phase III trials and nearly 300 in phase II trials.

Orphan Indication Pipeline

This expansive pipeline represents progress for patients and advances in scientific research, and also ensures that the industry-wide discussions surrounding approval pathways, reimbursement and patient-centric care will remain at the forefront.  The 2013 BIO International Convention in Chicago will host an Orphan Disease Forum featuring industry thought leaders discussing these issues and more.  For additional information on the Forum, which is taking place on April 23 and is sponsored by Genzyme, Alexion and CSL Behring, please visit this page.