Swedish Biotech Can Now Fast-Track its Metabolic Disorder Drug

Sobi will enter the clinic after the Investigational New Drug application for its rare metabolic disorder candidate was accepted by the FDA.

The Swedish Orphan Biovitrum (Sobi) will begin clinical trials for SOBI003, its candidate for the rare metabolic disorder, Sanfilippo syndrome type A. This was made possible as the FDA’s accepted the company’s Investigational New Drug (IND) application. In addition, the company’s candidate has received Fast Track status from the FDA, which will help the candidate move into the clinic in 2018.

Sanfilippo A syndrome is a life-threatening inherited metabolic disorder that affects children from a young age. It belongs to a group of diseases called Lysosomal Storage Disorders as the body cannot break down heparan sulfate, a long chain sugar molecules, which causes its buildup in lysosomes. This causes the severe progressive degeneration of the central nervous system. Sobi’s candidate is an enzyme replacement therapy for sulfamidase, which is delivered into lysosomes to reduce the storage of heparan sulfate.

The company will come up against Lysogene, a company set up by Karen Aich, a top female entrepreneur, after her daughter was diagnosed with the disease. This biotech has developed a gene therapy to replace the defective sulfamidase enzyme, which is undergoing a Phase II/III trial.


Image – Spectral-Design / shutterstock.com

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