Green Cross and Hanmi Pharmaceutical Submit Clinical Trial Plan in the US for Rare Disease 'Fabry Disease' Treatment

GC Green Cross and Hanmi Pharmaceutical announced on the 1st that they have submitted the clinical trial application (IND) for the Phase 1 and 2 clinical trials of ‘LA-GLA (development code GC1134A·HM15421)’, a treatment for Fabry disease, a type of lysosomal storage disorder, to the U.S. Food and Drug Administration (FDA).

GC Green Cross Headquarters Exterior [Photo by GC Green Cross]

LA-GLA is an innovative new drug for Fabry disease jointly developed by GC Green Cross and Hanmi Pharmaceutical as the world’s first once-monthly subcutaneous injection therapy. Fabry disease is a rare X-linked genetic disorder and a type of lysosomal storage disorder. Lysosomal storage disorders are caused by genetic defects that result in insufficient production of enzymes responsible for breaking down unnecessary substances in the body, leading to accumulation of these substances that damage cells and organs. Fabry disease occurs due to a deficiency of the enzyme alpha-galactosidase A, which breaks down glycolipids. Other representative diseases include Hunter syndrome (Mucopolysaccharidosis type II) and Sanfilippo syndrome (Mucopolysaccharidosis type III). In addition to LA-GLA, GC Green Cross has already globally launched Hunterase, a treatment for Hunter syndrome.

Lysosomal storage disorders, including Fabry disease, are treated with enzyme replacement therapy (ERT), which involves intravenous injection of enzymes developed through recombinant gene technology to replace the deficient enzymes. However, this treatment requires long intravenous infusions at the hospital every two weeks, posing treatment burdens related to intravenous administration and limitations such as insufficient efficacy in suppressing progressive kidney disease.

LA-GLA is positioned as a ‘next-generation long-acting enzyme replacement therapy’ that improves upon the limitations of existing treatments. Based on superior efficacy in improving kidney function, vascular disease, and peripheral neuropathy compared to existing treatments in preclinical stages, it was designated as an orphan drug (ODD) by the U.S. FDA last May.

A representative from GC Green Cross stated, “We plan to conduct global clinical trials including Korea, led by the United States,” and added, “We will do our best in new drug development to provide new treatment options for patients suffering from Fabry disease based on our experience, knowledge, and know-how in developing treatments for lysosomal storage disorders.”