In July, the U.S. Food and Drug Administration (FDA) granted orphan drug designation
to SOBI003 for the potential treatment of mucopolysaccharidosis type IIIA (MPS IIIA).
This morning, the developer of the drug, Sobi, announced that the FDA has issued a Study may proceed letter
for the first study in humans, thereby accepting the investigational new drug (IND) application for the product candidate, and the regulatory agency additionally granted SOBI003 Fast Track status.
MPS IIIA, commonly referred to as Sanfilippo syndrome type A, occurs in children with insufficient levels of specific enzymes essential for metabolizing heparan sulfate. This can lead to a build-up of long chains of sugar molecules in lysosomes and result in progressive neural degeneration. The disease is most often detected in patients around 4 years of age when they present with developmental delay and unexplained behavioral problems. Affected children are not expected to live beyond the third decade of life.
MPS IIIA progresses rapidly with intellectual decline, motor retardation, eventual dementia, and severe behavioral disturbances such as tantrums, aggressiveness and hyperactivity.
"We are very pleased with the IND acceptance and Fast Track status granted by the FDA,” said Milan Zdravkovic, Chief Medical Officer and Head of Research and Development at Sobi in a press release
. “It is an important step towards initiating the first clinical study with SOBI003 in children affected by MPS IIIA. The Fast Track status granted by the FDA is a milestone and acknowledgement of the significant unmet medical need that SOBI003 may be fulfilling.”
The clinical study of SOBI003 in patients with this rare metabolic disorder is expected to start later this year. The potential therapy utilizes Sobi’s one-of-a-kind glycan modification technology and is being developed as an enzyme replacement therapy with the intention of lessening heparin sulfate storage materials in affected cells. It is taken up by cells and transferred into the lysosomal compartment where heparin sulfate is damaged, and the reformation of the molecule manufactures a prolonged half-life.
"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", commented Dr. Paul Harmatz, UCSF Benioff Children's Hospital in Oakland California.
Currently, there are no approved treatments for MPS IIIA. An estimated 1,000-2,000 persons are living with MPS IIIA in the EU and U.S.
For more from the FDA, including applications, designations and approvals, follow Rare Disease Report