European orphan drug award for Agilis Bio therapeutics for AADC Deficiency

Release Date: 25-Jan-2017

Agilis Bio therapeutics Inc., a biotechnology company focused to develop DNA therapeutics for rare genetic diseases that affect the central nervous system (CNS) has recently declared that European Commission has granted Orphan Medicinal Product (OMP) designation in the European Union (EU) to the Company’s gene therapy product candidate, AGIL-AADC. In 2015, Agilis received Orphan Drug Designation for AGIL-AADC from the U.S. Food and Drug Administration (FDA). AGIL-AADC is an innovative gene therapy candidate being investigated to treat AADC deficiency by using an AAV vector to deliver a corrective DDC gene to rescue deficits in patients suffering from this disease.


AGIL-AADC is being developed for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency, a rare disease arising from mutations in the dopa decarboxylase (DDC) gene. AADC deficiency can result in severe developmental failures, global muscular hypotonia and dystonia, the need for life-long care and premature death. AGIL-AADC is the first therapeutic candidate to receive orphan designation for AADC deficiency in Europe.


Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel 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.  The OOPD provides incentives for sponsors to develop products for rare diseases, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. 


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