European Commission grants SOBI003 orphan designation for the treatment of MPS IIIA
Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO: SOBI) has been granted orphan designation by the European Commission (EC) for the company’s development product candidate SOBI003, a chemically modified human recombinant sulfamidase for the treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome). SOBI003 will be included in the EU Community Register of Orphan Medicinal Products. MPS IIIA or Sanfilippo A syndrome is an inherited systemic lysosomal storage disease with a significant central nervous system component. Onset is in early childhood and the