FDA grants SOBI003 Orphan Drug Designation for the treatment of MPS IIIA

Swedish Orphan Biovitrum has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the company’s development candidate SOBI003, a chemically modified human recombinant sulfamidase for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A, a rare metabolic disorder.

“We are very pleased with the Orphan Drug Designation from the FDA for SOBI003. MPS IIIA is a severe and debilitating disease, and this development program is an important part of our mission of being pioneers in rare diseases. We are currently in the late pre-clinical phase and expect to initiate the first clinical trial with SOBI003 in 2018,” said Milan Zdravkovic, Chief Medical Officer and Head of Research and Development at Sobi.

Onset of Mucopolysaccharidosis type IIIA is in early childhood, and the disease is characterised by severe and progressive developmental delay, motor retardation and eventually dementia. One in 100,000 children are born with the inherited condition and very few patients survive into adulthood. There is presently no treatment for MPS IIIA. SOBI003 was granted orphan designation by the European Commission for MPS IIIA in October 2016.