Novelpharma, a bio-venture specializing in rare medicines, is accelerating the development of a treatment for Sanfilippo Syndrome Type A (MPS IIIA).
On the 30th, Novelpharma announced that it has submitted an Investigational New Drug (IND) application for the Phase 1 clinical trial of ‘NP3011 (substance name GC1130A)’, an MPS IIIA treatment co-developed with GC Green Cross, to the Ministry of Food and Drug Safety (MFDS).
Starting with the IND application, a multinational clinical trial for NP3011 will commence within the year. On the 23rd, the company also applied for a Phase 1 IND and Fast Track designation from the U.S. Food and Drug Administration (FDA).
MPS IIIA is a recessive genetic disorder characterized by the accumulation of heparan sulfate in the body due to a gene defect. Novelpharma, together with GC Green Cross, is developing an MPS IIIA treatment that directly supplements the deficient enzyme in the central nervous system via intracerebroventricular (ICV) administration.
The treatment has been designated as an orphan drug in the United States and Europe. Last year, the company received Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the FDA. Subsequently, in January of this year, it was also designated as an orphan drug by the European Medicines Agency (EMA).
A company representative stated, "To intensify the development of our main MPS IIIA treatment, we have proceeded with the Phase 1 IND application domestically following the U.S. submission. We aim to achieve concrete results alongside the multinational clinical trial starting within the year."
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