Announced details
JR-171: CSF-HS concentrations decreased in all patients by Week 12 after treatment initiation
JR-171: treatment adherence exceeded 90%
JR-171: incidence of anaphylaxis and infusion-related reactions below 5%
JR-171: the 4.0 mg/kg high-dose group showed a greater reduction trend than the 2.0 mg/kg low-dose group
JR-171: 6 patients in the low-dose group and 8 in the high-dose group. JR-141: pooled data from 5 open-label studies, including follow-up of up to 5 years
FDA Fast Track designation for JR-171
U.S. Food and Drug Administration (FDA)
In this announcement the company addresses none of Contract Value / Offering price / Impact on Business Performance.
18th International Symposium on MPS and Related Lysosomal Diseases 2026
The 22nd WORLDSymposium™ 2026
Based on the Company's research
References 1–8
| Item | Details |
|---|---|
| Names of Products / Services Covered | JR-171（lepunafusp alfa）, JR-141（pabinafusp alfa）, IZCARGO® intravenous infusion 10 mg, J-Brain Cargo® platform technology |
| Launch date | IZCARGO® intravenous infusion 10 mg: marketed since May 2021 (actual performance) |
| Applicable area | Mucopolysaccharidosis type I (MPS I), Mucopolysaccharidosis type II (MPS II), Lysosomal diseases, Central nervous system symptoms |
| Award/certification name | FDA Fast Track designation for JR-171 |
| Awarding / certifying body | U.S. Food and Drug Administration (FDA) |
| Quantitative Effects | JR-171: CSF-HS concentrations decreased in all patients by Week 12 after treatment initiation, JR-171: treatment adherence exceeded 90%, JR-171: incidence of anaphylaxis and infusion-related reactions below 5%, JR-171: the 4.0 mg/kg high-dose group showed a greater reduction trend than the 2.0 mg/kg low-dose group |
| Usage history | JR-171: 6 patients in the low-dose group and 8 in the high-dose group. JR-141: pooled data from 5 open-label studies, including follow-up of up to 5 years |
| Source | 18th International Symposium on MPS and Related Lysosomal Diseases 2026, The 22nd WORLDSymposium™ 2026, Based on the Company's research, References 1–8 |
Items this document does not state (10)
These are items the company does not state in this document. We do not leave them blank, nor fill them in from other sources or by inference.
The reasons the company gives
The company's own explanation, not something inferred from the numbers.To share research findings at a forum promoting advances in research and medical care, early diagnosis, the development of treatments, and improved access to healthcare for mucopolysaccharidoses and related lysosomal diseases.