On the 23rd, Nobelpharma announced that it has submitted an Investigational New Drug (IND) application and a Fast Track Designation request to the U.S. Food and Drug Administration (FDA) for the clinical Phase 1 trial of the Sanfilippo Syndrome Type A treatment (MPS III A), which is being co-developed with GC Green Cross.
Fast Track is a program created by the FDA to accelerate drug development for serious diseases with unmet medical needs. Being designated as Fast Track provides the benefit of regular communication with the FDA.
With this IND and Fast Track application, the clinical development and new drug development of NP3011 (substance name GC1130A), the MPS IIIA treatment, are expected to accelerate. The company plans to conduct global clinical trials in the U.S. as the lead, as well as in Korea and Japan, and will evaluate the safety and tolerability of NP3011 through the Phase 1 trial.
MPS IIIA is a hereditary lysosomal storage disease (LSD) in which heparan sulfate accumulates in the central nervous system, including the brain and spinal cord, causing progressive damage to the central nervous system. There are currently no developed treatments, so patients rely on symptomatic therapies.
Nobelpharma, together with GC Green Cross, is developing a treatment that directly supplements the deficient enzyme in the central nervous system via intracerebroventricular (ICV) administration. This treatment was designated as a Rare Pediatric Disease Drug (RPDD) and an Orphan Drug (ODD) by the FDA last year. It also received orphan drug designation from the European Medicines Agency (EMA) in January.
A representative from Nobelpharma said, "With this IND application, we have taken the first step toward developing an innovative drug for Sanfilippo Syndrome Type A. Following the U.S., we will accelerate clinical trials in Korea and Japan to bring hope to patients suffering from Sanfilippo Syndrome Type A worldwide."
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