Swedish Orphan Biovitrum AB confirmed FDA’s acceptance of the investigational new drug application for the drug candidate SOBI003 along with granting Fast track status to it. SOBI003 is a chemically modified human recombinant sulfamidase for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA), a rare metabolic disorder also known as Sanfilippo syndrome type A.
Milan Zdravkovic, Chief Medical Officer and Head of Research and Development at Sobi said “We are very pleased with the IND acceptance and Fast Track status granted by the FDA. It is an important step towards initiating the first clinical study with SOBI003 in children affected by MPS IIIA. The Fast Track status granted by the FDA is a milestone and acknowledgement of the significant unmet medical need that SOBI003 may be fulfilling.”