Accept Cookies?
Provided by OpenGlobal E-commerce

Please wait while your page loads ...

FARAFARA Cure FA

Agilis Biotherapeutics Announces Orphan Product Designation Approval in Europe for the Treatment of Friedreich Ataxia

First Gene Therapy Treatment Candidate to Receive Orphan Designation in EU and USA

Agilis Biotherapeutics, Inc. (Agilis), a biotechnology company advancing innovative DNA therapeutics for rare genetic diseases that affect the central nervous system (CNS), announced today that the European Commission (EC) has granted Orphan Medicinal Product (OMP) designation in the European Union (EU) to the Company’s gene therapy product candidate, AGIL-FA, being developed for the treatment of Friedreich ataxia (FA), an inherited degenerative neuromuscular disorder resulting in loss of motor coordination and strength, hearing, vision, speech and often premature death. The EC’s approval follows a positive opinion in July 2017 from the European Medicine Agency’s (EMA) Committee for Orphan Medicinal Products (COMP). This follows the Orphan Drug Designation for AGIL-FA granted by the U.S. Food and Drug Administration (FDA) last year. The Company’s gene therapies for AADC deficiency and Angelman syndrome have previously received orphan status in both the EU and US.

"Receiving the first orphan designations for a gene therapy product candidate from the FDA and now the EU for the treatment of FA is an honor," said Mark Pykett, President and CEO of Agilis. "The orphan designation is another step on our path to bring this important new therapy to patients who currently lack treatment options."

Read the entire article HERE


About the Author

Jen Farmer

Jen Farmer

Executive Director

SHARE

FacebookTwitterLinkedinShare on Google+
Family A.jpg

 

Archived in
  Scientific News


 

 

Tagged in
FARA Scientific News