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Cushing’s Disease: FDA Orphan Drug Designations

November 28, 2014

This Blog Post presents the FDA designated orphan drugs for the rare disease Cushing’s Disease.

I – Introduction

Cushing’s Disease (CD) is not to be confused with Cushing’s Syndrome. Cushing’s Syndrome occurs when one’s body is exposed to high levels of the hormone cortisol. Cushing’s Syndrome can be caused by too many corticosteroid medications or can develop because one’s body produces too much cortisol. CD refers to a disorder where one’s pituitary gland releases too much Adrenocorticoltropoic Hormone (ACTH), which then signals to the adrenal glands to produce cortisol. In other words, too much ACTH can result in too much cortisol. CD is a form of Cushing’s Syndrome. CD is caused by a tumor or hyperplasia of the pituitary gland. The first line and most common treatment for CD is surgical removal of the tumor.

It was Dr. Harvey Cushing, an American neurosurgeon, who more than 80 years ago discovered that a tumor on the pituitary gland causes a hormone disorder, which became known as CD. Awareness of CD is low and patients often struggle with getting the right diagnosis for CD for many years.

II – FDA Orphan Drug Designations and Approvals for CD

The chart below identifies FDA Orphan Drug Designations (ODDs) and approvals for CD in ascending ODD Date order :

#   Generic Name/ODD Date Sponsor Company Indication Approval Date
1 Pasireotide (Signifor)/  07.24.09 Novartis Cushing’s Disease 12.14.12
2 Osilodrostat/  09.13.13 Novartis Cushing’s Disease N/A


Novartis in December 2012, receives FDA approval for orphan drug Signifor (Pasireotide) injection, for the treatment of adults with CD for whom pituitary surgery is not an option or has not been curative. Signifor is the first medication in the US to be approved that “addresses the underlying mechanism of CD.”

In April 2012, the European Commission approves Signifor for CD for adults where surgery is not an option or where surgery has failed.

Please Note:Chemlab2” by Tahoenathan (Own work) [CC-BY-SA-3.0] | via Wikimedia Commons.

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

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