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Swedish Orphan Biovitrum AB: European Commission grants SOBI003 orphan designation for the treatment of MPS IIIA

Swedish Orphan Biovitrum AB (publ) ( (Sobi™)
(STO: SOBI) 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

"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.


About orphan designation

To be granted orphan designation in the EU, the new investigational
product must be for the diagnosis, prevention or treatment of a
life-threatening or chronically debilitating condition that affects
no more than five in 10,000 people in the EU and for which no
satisfactory treatments exist or, where they do exist, where the new
medicine will be of significant benefit to those affected by that
condition. It can also be granted if a new product is not expected to
make a sufficient return to justify the investment.

About mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)

MPS IIIA or Sanfilippo A syndrome is a progressive, life-threatening
and rare inherited metabolic disorder affecting children already from
a young age. MPS IIIA belongs to a group of diseases called Lysosomal
Storage Disorders (LSDs). In MPS IIIA, the body is unable to break
down long chains of sugar molecules called heparan sulfate, resulting
in heparan sulfate accumulation in lysosomes. MPS IIIA mainly affects
the central nervous system where it causes severe progressive

Approximately 1,000-2,000 persons are estimated to live with MPS IIIA
in the EU and US. The disease is usually identified at around four
years of age and the life-span of an affected child does not usually
extend beyond the third decade of life. There is no treatment for MPS
IIIA to date.

About SOBI003

SOBI003 is a chemically modified variant of a recombinant human
sulfamidase product candidate 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, which
may enable transport of SOBI003 over the blood brain barrier and
facilitate uptake of SOBI003 in to the brain.

About Sobi™

Sobi™ is an international speciality healthcare company dedicated to
rare diseases. Our mission is to develop and deliver innovative
therapies and services to improve the lives of patients. The product
portfolio is primary focused on Haemophilia, Inflammation and Genetic
diseases. We also market a portfolio of speciality and rare disease
products across Europe, the Middle East, North Africa and Russia for
partner companies. Sobi is a pioneer in biotechnology with
world-class capabilities in protein biochemistry and biologics
manufacturing. In 2015, Sobi had total revenues of SEK 3.2 billion
(USD 385 M) and approximately 700 employees. The share (STO:SOBI) is
listed on NASDAQ OMX Stockholm. More information is available at

For more information please contact

Media relations Investor relations
Linda Holmström, Senior Jörgen Winroth, Vice
Communications Manager President, Head of Investor
+46 708 734 095 +1 347-224-0819, +1 212-579
-0506, +46 8 697 2135

Valstar et al. Ann Neurol. 2010;68(6):876-87

Swedish Orphan Biovitrum AB
Postal address SE-112 76 Stockholm, Sweden
Phone: +46 8 697 20 00


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