Dosage twice a week → once a month… Treatment convenience ↑ Two new drug research results announced at ‘World Symposium 2024’ Designated as a rare disease and clinical trials promoted based on excellent efficacy
Researchers Wonki Kim (left) and Chorong Park from the Hanmi Pharmaceutical R&D Center are presenting a poster on the main research results of the Fabry disease treatment drug ‘LA-GLA’ at the World Symposium 2024.
Hanmi Pharmaceutical and GC Green Cross announced the main results of their joint research on ‘the world’s first innovative new drug for treating Fabry disease in a once-monthly subcutaneous dosage form’ at a conference where rare disease experts from around the world gather. It received a lot of attention from rare disease researchers due to its superior efficacy compared to existing treatments.
Hanmi Pharmaceutical presented two posters showing the results of research confirming the excellent efficacy of ‘LA-GLA (development names HM15421, GC1134A)’ at the ‘WORLD Symposium 2024’ held in San Diego, USA from the 4th to the 9th. It was announced on the 15th that it was done. It is said to have shown better efficacy than existing treatments in improving kidney function, vascular disease, and peripheral nerve disorders.
Fabry disease is a rare disease inherited through sex chromosomes and is a type of lysosomal storage disease (LSD). It occurs due to a deficiency of the enzyme ‘alpha-galactosidase A’, which decomposes glycolipids in the ‘lysosome’, an intracellular organelle that removes unnecessary substances. As unprocessed glycolipids continue to accumulate in the body, cytotoxic and inflammatory reactions occur, resulting in various diseases. It is a progressive, incurable disease that gradually damages organs and leads to death.
Hanmi Pharmaceutical and GC Green Cross are said to have confirmed LA-GLA’s ‘stability in lysosomes’ and ‘extended in vivo half-life’ through the results of this study. Based on this, it was possible to present a new paradigm for treating Fabry disease. Currently, patients with Fabry disease are treated with enzyme replacement therapy (ERT), which involves intravenous administration of enzymes developed through genetic recombination technology. These first-generation treatments have the inconvenience of having to be administered intravenously for several hours at a hospital once every two weeks. In addition, it is known to have clear limitations, including the treatment burden due to intravenous infusion and lack of effectiveness against progressive kidney function deterioration.
LA-GLA, which is being jointly developed by Hanmi Pharmaceutical and GC Pharma, is a ‘next-generation long-acting enzyme replacement therapy treatment’ that improves the limitations of existing first-generation treatments. It is being developed as a subcutaneous administration once a month.
In this study, repeated administration of LA-GLA in an animal model of Fabry disease confirmed the efficacy of improving kidney function and fibrosis compared to existing treatments. It is said that repeated administration of LA-GLA significantly improved peripheral sensory function and histological lesions of the nerve cells that control it. Hanmi Pharmaceutical reported that the phenomenon of increased blood vessel wall thickness due to glycolipid accumulation was also effectively improved. Based on the results, Hanmi Pharmaceutical and GC Green Cross are preparing to apply for orphan drug designation (ODD) and clinical trial designation (IND) for LA-GLA.
An official from Hanmi Pharmaceutical said, “Considering the suffering experienced by patients and their families, the development of treatments for rare diseases is an area where pharmaceutical companies must maintain their original sense of mission and push forward to the end.” He added, “We have successfully developed a next-generation treatment for Fabry disease that presents a new treatment paradigm. “We will continue to focus our research capabilities to commercialize it,” he said.
Hanmi Pharmaceutical and GC Pharma signed a contract to jointly develop next-generation innovative new drugs for LSD treatment in 2020. Joint research is currently underway with the goal of developing global innovative new drugs in the field of rare diseases.
Meanwhile, the World Symposium is an academic society celebrating its 20th anniversary this year, and experts from all over the world gather to discuss research related to lysosomal diseases. It has become a global forum with more than 2,000 attendees from over 50 countries around the world.
Kim Min-beom, Donga.com reporter mbkim@donga.com
2024-02-15 13:40:00
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