Swedish Orphan Biovitrum has been granted orphan designation by the European Commission (EC) for the company’s development product candidate SOBI003, a chemically modified human recombinant sulfamidase for the treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome).

SOBI003 will be included in the EU Community Register of Orphan Medicinal Products.

MPS IIIA or Sanfilippo A syndrome is an inherited systemic lysosomal storage disease with a significant central nervous system component. Onset is in early childhood and the disease is characterised by severe and progressive development delay including behavioural problems, motor retardation and eventually dementia. Very few patients survive into their twenties. There is presently no treatment for MPS IIIA.

“MPS IIIA is a severe and debilitating disease with devastating consequences for patients. We hope that SOBI003 will provide these patients with a treatment where there is none today,” said Stephen James, Head of Research and Translational Sciences at Sobi.

Sobi is in the late stages of preclinical development of SOBI003. Preclinical studies to date with repeated systemic infusions have demonstrated efficacy in reducing substrate levels in the brain and signs of disease modifying effects.  Sobi is preparing for clinical studies, which it plans to start in 2018. Clinical studies will focus on exploring the safety and efficacy of SOBI003 in MPS IIIA patients.