GC Green Cross and Hanmi Pharmaceutical have successfully administered the first dose to a patient in the Phase 1/2 clinical trial of their jointly developed innovative new drug for Fabry disease, marking the official start of global clinical trials.
On May 15, GC Green Cross and Hanmi Pharmaceutical announced that the first administration of their jointly developed Fabry disease treatment, LA-GLA (code name GC1134A/HM15421), to a domestic patient has been successfully completed.
GC Green Cross and Hanmi Pharmaceutical have received IND (Investigational New Drug) approval for Phase 1/2 clinical trials in both the United States and Korea to conduct global clinical studies for LA-GLA. In addition, on April 21, they received IND approval for Phase 1/2 clinical trials in Argentina, where they plan to evaluate the safety, tolerability, pharmacokinetics, and efficacy of the drug in adults aged 18 and older diagnosed with Fabry disease.
This Phase 1/2 clinical trial is being conducted at a total of 10 institutions, including six sites such as the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) in the United States, Yangsan Pusan National University Hospital and Severance Hospital in Korea, and two institutions in Argentina. Patients deemed eligible for participation through screening will receive subcutaneous injections of LA-GLA once every four weeks.
Fabry disease is a progressive, rare, and intractable genetic disorder linked to the sex chromosomes and is classified as a type of Lysosomal Storage Disease (LSD). It occurs when there is a deficiency of the enzyme alpha-galactosidase A, which breaks down glycolipids in the lysosome, an organelle responsible for removing unnecessary substances from cells. The continuous accumulation of unmetabolized glycolipids in the body leads to cytotoxicity and inflammatory responses, gradually damaging various organs and potentially resulting in death in severe cases.
Currently, most Fabry disease patients are treated with enzyme replacement therapy (ERT), which involves intravenous infusion of enzymes developed using recombinant DNA technology.
However, these first-generation therapies have unresolved unmet medical needs due to several limitations: patients must undergo lengthy intravenous infusions at the hospital every two weeks, which is inconvenient; there are adverse effects associated with intravenous administration; and the progression of diseases in major organs, including the heart, kidneys, and both central and peripheral nervous systems, cannot be fully suppressed.
LA-GLA is attracting attention as a 'next-generation long-acting enzyme replacement therapy' that addresses the limitations of existing treatments. It significantly improves convenience by being administered as a subcutaneous injection once a month. Preclinical studies have demonstrated its superior efficacy in improving renal function, vascular disease, and peripheral neuropathy compared to existing therapies. Based on these results, it was designated as an Orphan Drug by the US Food and Drug Administration (FDA) in May last year, and as a rare drug in development by the Ministry of Food and Drug Safety (MFDS) in Korea in January this year.
Hong Guru, a professor at Sinchon Severance Hospital, stated, "I am very pleased that our hospital was able to administer the first dose in the global clinical trial. I hope this trial will provide a new treatment option for many Fabry disease patients."
Shin Sugyeong, Head of the Medical Division at GC Green Cross, said, "We are delighted that the global clinical trial of LA-GLA is officially underway with the first patient administration. With our experience in developing treatments for various lysosomal storage diseases such as MPSIIIA, GC Green Cross will do its utmost to provide new treatment options for Fabry disease patients."
Lee Moonhee, General Manager of Clinical Development at Hanmi Pharmaceutical, commented, "We hope that LA-GLA, which incorporates the 'LAPSCOVERY' technology that dramatically extends drug efficacy, will become a treatment that offers greater convenience and meaningful results for Fabry disease patients. Together with GC Green Cross, we will do our best in clinical development."
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