New Drug For Mucopolysaccharidosis Gets FDA Approval For Study

The FDA approved a human study to be conducted by Swedish Orphan Biovitrum AB to treating mucopolysaccharidosis type lllA patients with a new drug called SOB1003, reported European Pharmaceutical Review. Not only did the treatment development company receive that approval, but they also were deemed fast track status by the FDA as well, which shows the FDA’s faith in its potential.

Mucopolysaccharidosis type lllA, as known as MPS lllA and Sanfilippo syndrome type A, is a rare inherited metabolic disease. Diagnosis typically comes at a young age and the disease progressively gets worse over time. The body fails to break down sugar molecule chains (which are known as heparan sulfate), resulting in a unnatural buildup in lysosomes. The disease targets the central nervous system, leaving patients debilitated. It is often fatal– only a very small percentage of patients making it to their adult years. To learn more about this rare disease, click here.

The treatment they look to use in their study, SOBI003, uses Sobi’s top-of-the-line glycan modifying technology. It’s a therapy that replaces enzymes and aims to reduce the storage of heparan in the cells affected. They basically modify the molecule.

This disease has needed attention for a long time, as there is no known treatment to cure the disease, and little help to aid symptoms. Due to the need, Dr. Paul Harmatz from UCSF Benioff Children’s Hospital expressed how huge this is for mucopolysaccharidosis patients and their families. Additionally, The Chief medical officer of Sobi Development, Milan Zdravkovic shared his excitement with the acceptance and fast track. He said this is the first human clinical study with the treatment in children and believes it to be a huge milestone for the mucopolysaccharidosis community, especially considering the overdue need of medical attention. They anticipate the start of the studying taking place this year and couldn’t be more excited for what’s to come.

Do you know anyone with mucopolysaccharidosis? Share your stories, thoughts, and hopes, with the Patient Worthy community!

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