RARE Daily

FDA Grants Fast Track Designation to GCBP and Novel Pharma’s Experimental MPSIIIA Therapy

June 10, 2024

Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation to GC Biopharma and Novel Pharma for their jointly developed MPSIIIA experimental treatment, GC1130A.

MPSIIIA, also known as Sanfilippo syndrome Type A, is a rare genetic disorder that causes central nervous system damage through the accumulation of heparan sulfate, leading to progressive neurological decline. Without treatment, patients face life-threatening complications by the age of 15. GC1130A represents a potential breakthrough as the first and only treatment option specifically targeting MPSIIIA.

GC1130A is a biological drug that is being developed using high-concentration protein formulation technology, designed for administration to the central nervous system. It is delivered directly into the brain’s ventricles through intracerebroventricular injection, a method first applied globally by GC Biopharma’s Hunter syndrome treatment Hunterase, which has received market approval in Japan.

“This designation will enable us to accelerate the development of this new drug, bringing hope to patients and families affected by Sanfilippo syndrome,” GC Biopharma and Novel Pharma said.

GC Biopharma and Novel Pharma are preparing to initiate a multinational first-in-human clinical trial to evaluate the safety and tolerability of GC1130A in Korea, the United States, and Japan.

U.S. FDA’s Fast Track program is designed to expedite the development and review of drugs intended to treat serious or unmet medical needs. Fast Track designation provides extensive support, including frequent meetings with the FDA throughout the drug development, clinical, and approval stages.

The potential of GC1130A to meet the unmet medical needs of Sanfilippo syndrome has been recognized by major drug regulatory agencies; in 2023, the FDA granted GC1130A Rare Pediatric Disease and Orphan Drug designations, and earlier this year, the European Medicines Agency also granted GC1130A Orphan Drug status.


Stay Connected

Sign up for updates straight to your inbox.