Go to main content

NEWS

The latest news
Updates on GC Pharma’s
Journey of supporting
Health and happiness.

소식 상세 테이블 표
Title European Medicines Agency (EMA) Grants Orphan Drug Designation (ODD) to GC Biopharma's Treatment for Sanfilippo Syndrome (Type A)
Date of registration 2024-01-23
Attachment File
Contents

YONGIN, South Korea, January 23, 2024 - GC Biopharma Corp. (006280.KS) announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its intracerebroventricular (ICV) Enzyme Replacement Therapy (ERT) candidate, GC1130A, designed for Sanfilippo Syndrome type A (mucopolysaccharidosis type IIIA), developed in collaboration with Novel Pharma. 


GC1130A has previously achieved notable milestones by securing both Rare Pediatric Disease designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. FDA in January 2023. Building on these accomplishments, the latest approval in Europe adds another significant achievement. 


Sanfilippo Syndrome (type A) is a genetic disorder that triggers central nervous system damage by accumulating Heparan sulfate, leading to a progressive neurodegeneration in pediatric population. Patients with MPS IIIA typically have a life expectancy of approximately 15 years, and as of now, there are no approved therapies available. In nonclinical studies, GC1130A demonstrated both safety and efficacy in eliminating Heparan sulfate and restoring brain function. 


Since 2020, GC Biopharma and Novel Pharma have collaborated on the development of GC1130A. Utilizing proprietary recombinant protein manufacturing technology, GC Biopharma has successfully formulated GC1130A to be compatible with cerebrospinal fluid at high protein concentrations. Novel Pharma, a biotech company with expertise in rare disease drug development in Korea. 


"This EMA designation further underlines the potentials of our collaborative pipeline in addressing the disease pathology in upcoming clinical trials.", said GC Biopharma and added that it will be “committing to expeditiously advancing into clinical trials, acknowledging the urgent, unmet medical needs of Sanfilippo Syndrome patients.”


About GC Biopharma Corp.


GC Biopharma Corp. (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma Corp. is one of the leading plasma protein and vaccine product manufacturers globally and has been dedicated to quality healthcare solutions for more than half a century.


This press release may contain biopharmaceuticals in forward-looking statements, which express the current beliefs and expectations of GC Biopharma's management. Such statements do not represent any guarantee by GC Biopharma or its management of future performance and involve known and unknown risks, uncertainties and other factors. GC Biopharma undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule.


List view

이메일집단수집거부

GC녹십자는 이메일 수집을 거부합니다

본 웹사이트에 게시된 이메일 주소가 전자우편 수집 프로그램이나
그 밖의 기술적 장치를 이용하여 무단으로 이메일 주소를 수집하는 행위를 거부하며,
이를 위반시 정보통신망 이용촉진 및 정보보호 등에 관한 법률에 의해 형사처벌됨을
유념하시기 바랍니다

게시일: 2003년 9월 1일