Search:
 
This image is of a spacer graphic
NHF Face Book NHF Twitter
+ Login to my NHF
+ NHF Membership
+ Donate to NHF
+ Chapter Center
+ Hechos y Respuestas Rápidas
+ Ethics Advisory Committee
This image is of a spacer graphic
-News
 NHF In The News
 NHF eNotes
 Medical Advisories
 Advocacy and Legislative Updates
 Medical News
 Blood Safety News
 NHF and Community News
-Industry News
 Travel Advisory

 

 

 
FDA Grants Orphan Drug Status to Alnylam’s Hemophilia Therapy
 

In August, Alnylam Pharmaceuticals, Inc., announced that the US Food and Drug Administration (FDA) had granted Orphan Drug Designation to ALN-AT3 for the treatment of hemophilia A. The company, based in Cambridge, Massachusetts, is developing ALN-AT3, a subcutaneously administered (injection just under the skin) RNAi therapy that targets antithrombin (AT) as a way to treat hemophilia A or B, hemophilia A or B with inhibitors, and other rare bleeding disorders. AT is a small plasma protein molecule that inactivates factor Xa and thrombin, which are needed for blood clotting.

 

ALN-AT3 incorporates Alnylam’s proprietary gene-silencing technology called RNAi, or RNA interference. Discovered by scientists in the late 1990s, RNAi is a natural process in which cells turn off, or silence, the activity of specific genes. ALN-AT3 silences certain genes associated with AT generation, “switching off” the protein’s production.

 

At the XXIV Congress of the International Society on Thrombosis and Haemostasis, June 29-July 4, in Amsterdam, Alnylam shared preclinical data from animal trials. The studies revealed that ALN-AT3 improved thrombin generation in mice and nonhuman primates.

 

Alnylam plans to file an investigational new drug application for ALN-AT3 in late 2013. It will initiate a Phase I clinical trial in humans in early 2014.

 

“We are very pleased that the FDA has granted Orphan Drug Designation for ALN-AT3 now for both the treatment of hemophilia A and hemophilia B. As a subcutaneously delivered RNAi therapeutic, we believe it represents an innovative approach for the management of hemophilia and has great potential to make a meaningful impact in the treatment of this often debilitating bleeding disorder,” said Saraswathy (Sara) Nochur, PhD, Senior Vice President, Regulatory Affairs and Quality Assurance at Alnylam. “ALN-AT3 is a key program in our ‘Alnylam 5x15’ product development and commercialization strategy, and we look forward to advancing this promising RNAi therapeutic into the clinic in the months to come.”

 

Sources: The Wall Street Journal, August 14, 2013, and Alnylam news release dated August 20, 2013