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Alnylam Pharmaceuticals, Inc.
, a leading RNAi therapeutics
company, announced today that the U.S. Food & Drug Administration (FDA) has
granted an Orphan Drug Designation (ODD) to ALN-AT3 as a therapeutic for the
treatment of hemophilia A. As reported last week, the FDA has also granted ODD
to ALN-AT3 for the treatment of hemophilia B. Alnylam is developing ALN-AT3, a
subcutaneously administered RNAi therapeutic targeting antithrombin (AT), for
the treatment of hemophilia – including hemophilia A, hemophilia B, and
hemophilia A or B with “inhibitors” – and other Rare Bleeding Disorders (RBD).
“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, Ph.D., 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.”
At the recent Congress of the International Society on Thrombosis and
Haemostasis, Alnylam presented pre-clinical data demonstrating that ALN-AT3
can normalize thrombin generation and improve hemostasis in hemophilia mice
and can fully correct thrombin generation in a non-human primate (NHP)
hemophilia “inhibitor” model. ALN-AT3 utilizes the company's proprietary
GalNAc conjugate delivery platform, enabling subcutaneous dose administration.
Alnylam plans to file an investigational new drug (IND) application for
ALN-AT3 in the fourth quarter of 2013 and initiate a Phase I clinical trial in
early 2014.
The FDA Office of Orphan Products Development (OOPD) mission is to advance the
evaluation and development of products that demonstrate promise for the
diagnosis and/or treatment of rare diseases or conditions. OOPD provides
incentives for sponsors to develop products for rare diseases. The Orphan Drug
Designation program provides orphan status to drugs and biologics which are
defined as those intended for the safe and effective treatment, diagnosis or
prevention of rare diseases/disorders that affect fewer than 200,000 people in
the U.S.
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