SOBI003 Gains Orphan Drug Status for Rare Metabolic Disorder

There is currently no approved treatment for Sanfilippo syndrome type A, which affects patients in early childhood.
There is currently no approved treatment for Sanfilippo syndrome type A, which affects patients in early childhood.

Sobi announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation to their developmental candidate for the rare metabolic disorder, mucopolysaccharidosis type IIIA (MPS IIIA). 

There is currently no approved treatment for Sanfilippo syndrome type A, which affects patients in early childhood. The progressive, life-threatening disorder manifests as severe and progressive developmental delay, motor retardation, and subsequent dementia. 

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SOBI003 is a chemically modified variant of a recombinant human sulfamidase candidate. MPS IIIA results in the accumulation of heparan sulfate in lysosomes due to the patient's inability to break down long chains of sugar molecules. Through the proprietary glycan modification technology, SOBI003 acts as an enzyme replacement therapy to reduce heparin sulfate storage materials in affected cells.

According to the Company, SOBI003 is currently in the late pre-clinical phase and its first clinical trial is expected to commence in 2018.

For more information visit Sobi.com.