SOBI003 Granted Orphan Drug Designation for MPS IIIA

Mathew Shanley

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to SOBI003, currently in development by Swedish Orphan Biovitrum AB (Sobi).
SOBI003 is a chemically-modified human recombinant sulfamidase for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA). MPS IIIA, commonly referred to as Sanfilippo syndrome type A, is a rare metabolic disorder and in it, heparan N-sulfatase is the affected enzyme.
Patients with MPS IIIA, or Sanfilippo syndrome type A, are characteristically children with inadequate levels of specific enzymes necessary to metabolize heparan sulfate. This can result in a build-up of these long chains of sugar molecules in lysosomes, leading to progressive neural degeneration. The disease is usually detected around 4 years of age when patients present with developmental delay and unexplained behavioural problems. The disease rapidly progresses with intellectual decline, motor retardation, eventual dementia, and severe behavioral disturbances such as tantrums, aggressiveness and hyperactivity. 
FDA Orphan Drug Designations provide an orphan status to drugs defined as a safe and effective treatment for rare diseases and disorders that affect fewer than 200,000 people in the U.S. At present, an estimated 1 in 100,000 children are born with the genetic condition MPS IIIA, and only a select few survive into adulthood, as the life-span of an affected child doesn’t typically extend past 30 years.
“We are very pleased with the Orphan Drug Designation from the FDA for SOBI003,” said Milan Zdravkovic, Chief Medical Officer and Head of Research and Development at Sobi via press release. “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.”
SOBI003 uses Sobi’s exclusive glycan modification technology and is intended to be treated as an enzyme replacement-type of therapy, lessening heparin sulfate storage materials in affected cells. It is taken up by cells and transferred into the lysosomal compartment where heparin sulfate is degraded, and the modification of the molecule produces a prolonged half-life.
“We are currently in the late pre-clinical phase and expect to initiate the first clinical trial with SOBI003 in 2018,” said Zdrakovic.
There is currently no approved treatment for MPS IIIA, and SOBI003 was also granted orphan designation by the European Commission for the disease in 2016.

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