Orchard Therapeutics’ (NASDAQ:ORTX) OTL-103 received FDA regenerative medicine advanced therapy (RMAT) designation for the treatment of Wiskott-Aldrich Syndrome (WAS).
WAS is an inherited immune disorder where autoimmunity and abnormal platelet function results in severe infections and bleeding episodes that can be life-threatening. OTL-103 is an ex vivoautologous hematopoietic stem cell gene therapy.
“Securing RMAT designation for OTL-103 is an important step in expediting the product development and review of our planned biologics license application and recognizes the unmet need for children and young adults afflicted with WAS,” Anne Dupraz-Poiseau, Orchard’s chief regulatory officer, said in a statement.
“We remain on track to file for regulatory approval of our WAS gene therapy program in the U.S. and Europe in 2021 in the hope of bringing this potentially curative therapy to patients as soon as possible,” she added.
Orchard recently reported positive interim results from the registrational trial for OTL-13 for WAS, and intends to present the full registrational trial data set later this year.
