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▎The content team editor of WuXi AppTec recently, JCR Pharmaceuticals announced that the Ministry of Health, Labour and Welfare (MHLW) of Japan has approved the listing of Izcargo (pabinafusp alfa) for the treatment of mucopolysaccharidosis type II (MPS II, also known as Hunter syndrome).
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Izcargo is a recombinant iduronic acid sulfatase (IDS) replacement therapy.
It is unique in that it uses the J-Brain Cargo delivery system developed by JCR.
This system can help intravenously injected biological products to cross the blood-brain barrier (BBB) and deliver them to the patient's brain.
The press release pointed out that this is the world's first enzyme replacement therapy that has been approved by regulatory agencies to cross the blood-brain barrier.
It may bring revolutionary changes to the lives of patients with lysosomal storage diseases.
Nature Reviews Drug Discovery today specifically reported on this progress.
Due to genetic mutations in MPS II patients, the key metabolic protease is defective or missing, which causes the accumulation of toxic metabolites in the lysosome.
Many patients have severe symptoms in the central nervous system (CNS), leading to poor neurocognitive development of the patients, affecting their independence and quality of life.
Although traditional enzyme replacement therapy can alleviate the patient's symptoms in other parts of the body, the molecular weight is too large to cross the blood-brain barrier that protects the brain, so it fails to effectively solve the patient's central nervous system symptoms.
Izcargo is a recombinant fusion protein that fuses IDS protease with an antibody targeting human transferrin receptor, and uses transferrin receptor-mediated transcytosis to help cross the blood-brain barrier.
Improve the level of Izcargo in the brain.
The approval of Izcargo by the Japanese Ministry of Health, Labour and Welfare is based on comprehensive evidence from preclinical and clinical studies.
In a phase 2/3 clinical trial conducted in Japan, the levels of biomarkers related to CNS diseases in the cerebrospinal fluid of all 28 patients were significantly improved, reaching the primary endpoint of the trial.
The trial also confirmed that 21 of 28 patients had maintained or improved their neurocognitive assessment.
"Izcargo's approval in Japan is an important milestone for the company.
It has obtained fast-track qualification from the US FDA, orphan drug qualification from the European Union EMA, and has been approved by the FDA to conduct phase 3 clinical trials in the United States.
" Shin Shin, president of JCR Pharmaceuticals Mr.
Ashida said: "We will continue to work to bring this new treatment option for lysosomal storage disease to patients all over the world as soon as possible.
" References: [1] JCR Pharmaceuticals Announces Approval of IZCARGO® (Pabinafusp Alfa) for Treatment of MPS II (Hunter Syndrome) in Japan.
Retrieved April 13, 2021, from %AE-Pabinafusp-Alfa-for-Treatment-of-MPS-II-Hunter-Syndrome-in-Japan Note: This article aims to introduce the progress of medical and health research, not a treatment plan recommendation.
If you need guidance on treatment plans, please go to a regular hospital for treatment.