FDA Designates DTX101 Orphan Drug for Hemophilia B
Dimension Therapeutics announced that the Food and Drug Administration (FDA) granted Orphan Drug designation to DTX101 for the treatment of moderate-severe to severe hemophilia B.
Dimension expects to initiate a multi-center phase 1/2 study to evaluate DTX101 in adult patients with moderate-severe to severe hemophilia B by the end of 2015.
DTX101 is designed to deliver blood clotting Factor IX (FIX) gene expression in patients with hemophilia B, a rare genetic bleeding disorder resulting from a deficiency in FIX.
For more information call (617) 401–0011 or visit DimensionTx.com.