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FDA Breakthrough Therapy Designation For An Orphan Drug: 26 And Counting

October 24, 2013

On October 24, the FDA grants Breakthrough Therapy Designation (BTD) #26 to Alexion Pharma International Sarl, a subsidiary of Alexion Pharmaceuticals. The FDA BTD is for orphan drug Cyclic Pyranopterin Monophosphate (cPMP or ALXN1101) for  the treatment of the ultra-rare disease, Molybdenum Cofactor Deficiency Type A (MoCD). cPMP, an enzyme co-factor replacement therapy, receives:

•   FDA orphan drug designation in November 2009

•   European orphan designation in September 2010.

MoCD Type A is a genetic metabolic disorder that affects newborns. It is caused by the lack of a substance called “molybdenum cofactor”, which is required for the production of several enzymes. Without the production of these enzymes, the toxic chemical sulfite will accumulate in the brain. Symptoms appear from birth and include seizures, an increase in reflex responses, feeding problems, and bleeding in the brain. MoCD is a life-threatening condition, with death usually occurring within a few months after birth. There is currently no treatment options for MoCD Type A. Alexion Pharmaceuticals has started a natural history study in MoCD Type A patients and has also completed dosing with the synthetic cPMP in a study in healthy volunteers.

This is the 2nd BTD for Alexion Pharmaceuticals – both for orphan drugs for ultra-rare diseases :

Row Num

Drug Name



Asfotase Alfa Hypophosphatasia (HPP)


Cyclic     Pyranopterin Monophosphate (cPMP) Molybdenum   Cofactor Deficiency (MoCD) Type A

The Column in the Chart titled “Drug Name” contains the URL for the link to the Press Release announcing the FDA Breakthrough Therapy Designation for each drug.

Please Note: FDA Official Logo from FDA website.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.


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