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.