SOBI003 Granted Orphan Drug Designation for MPS IIIA Treatment

Swedish Orphan Biovitrum AB (Sobi) announced that FDA has granted orphan drug designation to its development candidate SOBI003 for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A, which is a rare metabolic disorder. SOBI003 is a chemically modified variant of a recombinant human sulfamidase product candidate.

The onset of MPS IIIA typically begins in early childhood. One in 100,000 children are born with the inherited condition and very few patients survive into adulthood. MPS IIIA is characterized by severe and progressive developmental delay, motor retardation, and eventually dementia. There is currently no treatment for the disease.

SOBI003 is made using Sobi’s proprietary glycan modification technology. It is intended as an enzyme replacement therapy to reduce heparan sulfate storage materials in affected cells. SOBI003 is taken up by cells and transported into the lysosomal compartment where heparan sulfate is degraded. The modification of the molecule results in an extended half-life.

 “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 preclinical 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, in a press release.

SOBI003 was granted orphan designation by the European Commission for MPS IIIA in October 2016.

Source: Sobi