Sobi’s Enzyme Replacement Therapy for Sanfilippo Syndrome Type A to Enter Clinical Trials

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by Alice Melão |

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The U.S. Food and Drug Administration (FDA) has accepted an investigational new drug (IND) application for Swedish Orphan Biovitrum AB‘s (Sobi) enzyme replacement therapy SOBI003, for the treatment of Sanfilippo syndrome type A. The FDA also granted the therapy fast track status, which is expected to accelerate its clinical development and regulatory review, the company announced.

An IND is a request for FDA authorization to administer an investigational therapy to humans. It is generally the result of a successful preclinical development program and is the vehicle through which a sponsor advances an investigational therapy to clinical trials.

“We are very pleased with the IND acceptance and Fast Track status granted by the FDA. It is an important step towards initiating the first clinical study with SOBI003 in children affected by MPS IIIA,” Milan Zdravkovic, chief medical officer and head of research and development at Sobi, said in a press release. “The Fast Track status granted by the FDA is a milestone and acknowledgement of the significant unmet medical need that SOBI003 may be fulfilling,” he added.

Sanfilippo syndrome type A, also called mucopolysaccharidosis type IIIA (MPS IIIA), is a rare disorder caused by deleterious mutations in the SGSH gene, which encodes the sulfamidase enzyme. This enzyme is involved in the breakdown of large molecules known as glycosaminoglycans, which in the absence of the enzyme will accumulate inside cells. Glycosaminoglycans are believed to affect the activity of other proteins, and consequently, disrupt the normal functioning of cells. However, it is still unclear why this mechanism mostly affects nerve cells.

SOBI003 is a chemically modified human recombinant sulfamidase designed by Sobi to be a candidate for enzyme replacement therapy, with improved stability to overcome the limitations caused by SGSH mutations in Sanfilippo syndrome. SOBI003 is taken up by cells and transported into the area where glycosaminoglycans accumulate. This allows for the degradation of toxic molecules and the recovery of cellular balance.

“MPS IIIA is a life-threatening condition and very few patients survive into adulthood. There are huge unmet medical needs for these patients, since there is no disease modifying treatment available today,” said Paul Harmatz, an expert in gastroenterology, hepatology, and nutrition at UCSF Benioff Children’s Hospital in Oakland, California.

In October 2016, SOBI003 was granted orphan drug designation by the European Commission, and later by the FDA for Sanfilippo syndrome type A.

The clinical trial with SOBI003 is expected to begin during 2018.