EC grants drug orphan designation for CTCL



Micrograph showing
mycosis fungoides
The European Commission (EC) has granted orphan designation to MRG-106 for the treatment of cutaneous T-cell lymphoma (CTCL). MRG-106 is a locked nucleic acid-modified oligonucleotide inhibitor of miR-155-5p. miRagen Therapeutics, Inc., the company developing MRG-106, is currently testing the drug in a phase 1 trial of CTCL patients. Early... [Read Article]

EMA recommends drug receive orphan designation for PNH



Red blood cells
The European Medicines Agency (EMA) has recommended orphan drug designation for the complement C3 inhibitor APL-2 as a treatment for paroxysmal nocturnal hemoglobinuria (PNH). APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b, blocking all 3 pathways of complement activation... [Read Article]