2024-02-15 22:02:35
Sanofi-Aventis Korea’s Fabry disease treatment ‘Fabrazyme’ product image. Screenshot of homepage.
The development of a treatment for Fabry disease by Hanmi Pharmaceutical and GC Green Cross is on track. Both companies aim to develop new drugs with longer-lasting effects than existing treatments.
Hanmi Pharmaceutical and GC Green Cross conducted a non-clinical trial of ‘LA-GLA (code name HM15421/GC1134A)’, a Fabry disease treatment candidate, at the ‘WORLD Symposium 2024’ held in San Diego, USA from the 4th to the 9th of this month (local time). Two research results were presented as posters.
The two companies signed a contract to jointly develop innovative new drugs for lysosomal storage disease (LSD) in 2020. Disclosing the results of non-clinical research regarding 4 years following their development cooperation, the two companies revealed their specific goal of ‘developing a treatment for fibri disease that can be administered subcutaneously once a month.’
Fabry disease is a rare hereditary metabolic disease that is caused by a deficiency of the enzyme alpha-galactosidase A (α-gal A), which breaks down glycolipids. As a result, glycolipids such as globotriaosylceramide (Gb3) accumulate in the body, interfering with the function of cells and tissues and damaging organs in the long term.
One of the main treatment strategies for Fabry disease is enzyme replacement therapy (ERT), which supplements deficient enzymes by injecting artificially recombinant enzymes into the body, including Sanofi’s ‘Fabrazyme (ingredient name: Agalsidase beta)’ and Takeda’s ‘Fabrazyme’ (ingredient name: Agalsidase beta). ‘Lepragal (ingredient name: Agalsidase alfa)’ has been approved. Both drugs are administered intravenously.
The two domestic companies said that with existing enzyme replacement therapy treatments, there is the inconvenience of having to go to the hospital every two weeks and receive intravenous injections for several hours, and that LA-GLA improves kidney function, vascular disease, He emphasized that it was confirmed to have excellent efficacy in improving peripheral nerve disorders.
They announced that they would overcome the limitations of existing drugs by targeting Fabrazyme, which can be considered a representative drug for Fabry disease. In the case of Fabrazyme, patients must visit the hospital once every two weeks and receive the treatment intravenously for regarding 4 to 5 hours.
It is also agreed in the medical field that the current medication cycle is a burden to patients. In particular, because it is a rare disease treatment, patients with rare diseases outside the metropolitan area have to travel long distances to local medical institutions to receive the treatment.
However, competition among drugs to treat Fabry disease is still fierce. In the case of Lepragal, which was released later than Fabrazyme in Korea, it is emphasized that it is derived from a human cell line rather than an animal cell line, and that the patient’s immunogenic response is low, so pretreatment is simple and the administration time is as short as 40 minutes. It is touted as an advantage.
On the other hand, long-term clinical studies have proven the long-term protective effect and safety profile of Fabrazyme, and there are claims that Fabrazyme, which is administered at a higher dose than Lefragal, is effective in reducing glycolipid accumulation that causes Fabry disease symptoms.
Although it is not an enzyme replacement therapy, an oral Fabry disease treatment that has significantly improved the convenience of administration has also been developed. ‘Galafold (ingredient name: migalastat)’, an oral Fabry disease treatment introduced by Handok in Korea, can be used on patients with compliant mutations, and the domestic prescription period is up to 60 days.
There have been previous cases where domestic companies succeeded in developing biosimilars for Fabry disease. Isu Abxis, an affiliate of Isu Group, has succeeded in commercializing ‘Fabagal’, a Fabrazyme biosimilar, and is supplying the product domestically and internationally. Isu Abxis announced that it achieved domestic sales of KRW 10.2 billion last year due to an increase in domestic prescription patients.
Despite existing prescription competition, high market growth rate is expected to serve as a driving force for new drug development for both companies. According to global market research firm Eveluate Pharma, the Fabry disease treatment market is expected to grow at an annual rate of 9.6% from $1.9 billion (approximately KRW 2.5337 trillion) in 2023 to $3 billion (approximately KRW 4 trillion) in 2028. .
Hanmi Pharmaceutical and GC Green Cross plan to apply for orphan drug designation (ODD) and enter phase 1 clinical trials within the year, and the industry is interested in which company will lead the clinical trials. At this point, both companies are reluctant to comment. Hanmi Pharmaceutical’s position is that it is difficult to disclose the matter as it is contract-related.
A GC Green Cross official said, “The R&R (roles and responsibilities) of both companies will be drawn in the future.”
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