Approval and Application Milestones for the Development Pipeline
First written in FY2016. 46 of 56 entries say "Advanced", first in FY2017-Q1. Latest entry: 2026-09-23.
7 other companies' latest entry also says "Advanced".
- 2026-09-23Advancedfinance.yahoo.co.jp
「Announced on 9/11 that dosing had begun in the global Phase I/II clinical trial of JR-446.」
It was reported that JR-446 had advanced to the dosing initiation stage of its global Phase I/II clinical trial. - FY2026AdvancedAnnual report
「Enrollment in JR-141's global Phase III clinical trial was completed ahead of schedule, and the company is currently working intensively toward global marketing approval applications.」
JR-141 achieved enrollment of the target number of patients in its global Phase III trial and held discussions with the FDA regarding its regulatory submission strategy. JR-142 completed patient enrollment in its Phase III trial, while JR-441 completed enrollment of the target number of patients in its Phase I/II trial in Germany and Phase I trial in Japan. JR-446's Phase I/II trial is continuing, and JR-471 has launched a clinical study to collect natural-history data. - FY2025AdvancedAnnual report
「The global Phase III clinical trial of pabinafusp alfa, a blood-brain barrier-penetrating enzyme replacement therapy for Hunter syndrome (development code: JR-141), is currently under way. Enrollment of the target 20 patients in Cohort B, which consists of patients with the so-called mild form of the disease, has been completed, and more than 95% of the patients in Cohort A, which consists of more severely affected patients, have also been enrolled. The Phase I/II clinical trial of the blood-brain barrier-penetrating enzyme replacement therapy for mucopolysaccharidosis type IIIA (development code: JR-441) is under way in Germany, where enrollment of the planned 12 patients was completed in the first half of 2024. In Japan, dosing of the investigational product began in the Phase I clinical trial in October 2024. For the blood-brain barrier-penetrating enzyme replacement therapy for mucopolysaccharidosis type IIIB (development code: JR-446), dosing began in the Phase I/II clinical trial in Japan in December 2024. An extension study of the Phase II clinical trial of the long-acting recombinant human growth hormone product redalsomatropin alfa (development code: JR-142) is under way. In Japan, dosing began in the Phase III clinical trial in December 2024.」
JR-141 completed enrollment of the target 20 patients in Cohort B, while Cohort A progressed to more than 95% enrollment. JR-441 completed enrollment of the planned 12 patients in its Phase I/II trial in Germany and began dosing in its Phase I trial in Japan; JR-446 also began dosing in its Phase I/II trial in Japan. JR-142 progressed to the dosing stage of its Phase III trial in Japan. - FY2025-H1AdvancedAnnual report
「An extension study of the Phase II clinical trial of the long-acting recombinant human growth hormone product (development code: JR-142) is under way. In July 2024, PMDA accepted the clinical trial notification for the Phase III trial, and preparations are currently under way to launch the trial.」
JR-142 is conducting an extension study of its Phase II trial, and PMDA accepted the clinical trial notification for the Phase III trial in July 2024. - FY2025-H1AdvancedAnnual report
「For the fucosidosis treatment, a lysosomal disease therapy applying J-Brain Cargo® (development code: JR-471), an exclusive license with sublicensing rights was granted to Medipal Holdings Corporation under the license agreement concluded in October 2022, covering commercialization activities, including research and development, manufacturing and sales, worldwide excluding Japan. Necessary research and other work is currently under way to launch clinical trials.」
Following the grant of an exclusive worldwide license, excluding Japan, to Medipal Holdings, JR-471 has progressed to the research stage ahead of the launch of clinical trials. - FY2025-H1AdvancedAnnual report
「For the blood-brain barrier-penetrating enzyme replacement therapy for mucopolysaccharidosis type IIIB (development code: JR-446), the clinical trial notification submitted in May 2024 was accepted by the Pharmaceuticals and Medical Devices Agency, and preparations are currently under way to launch the Phase I/II clinical trial.」
PMDA accepted the clinical trial notification for JR-446 submitted in May 2024, and the program progressed to preparations for the Phase I/II trial launch. - FY2025-H1AdvancedAnnual report
「The Phase I/II clinical trial of the blood-brain barrier-penetrating enzyme replacement therapy for mucopolysaccharidosis type IIIA (development code: JR-441) is under way in Germany. Enrollment of the planned 12 patients was completed in the first half of 2024. In Japan, dosing of the investigational product began in the Phase I clinical trial in October 2024.」
JR-441's Phase I/II trial is progressing in Germany, where enrollment of the planned 12 patients was completed in the first half of 2024. In Japan, dosing began in the Phase I trial in October 2024. - FY2025-H1ReframedAnnual report
「For this product, the policy is to advance development through an out-licensing arrangement rather than in-house development, and negotiations are under way with prospective partners regarding the license.」
For JR-171, the stated policy changed from continuing in-house development to advancing development through out-licensing, and the program reached the stage of negotiations with prospective partners regarding the license. - FY2025-H1AdvancedAnnual report
「The global Phase III clinical trial of pabinafusp alfa, a blood-brain barrier-penetrating enzyme replacement therapy for Hunter syndrome (development code: JR-141), is currently under way. Enrollment of the target 20 patients in Cohort B, which consists of patients with the so-called mild form of the disease, has been completed, and enrollment of more than 60% of the patients in Cohort A, which consists of more severely affected patients, has also been completed.」
In JR-141's global Phase III trial, enrollment of the target 20 patients in Cohort B was completed, while enrollment in Cohort A progressed to more than 60%. - FY2024Updated expected completion date for clinical trial enrollmentAnnual report
「For pabinafusp alfa, a blood-brain barrier-penetrating enzyme replacement therapy for Hunter syndrome (development code: JR-141), the product received Rare Pediatric Disease designation from the US Food and Drug Administration (FDA) in December 2022. In the global Phase III clinical trial, in which dosing of the first subject began in February 2022, efforts are under way to complete enrollment by the end of the first quarter of fiscal 2024 for the number of patients required for the interim analysis in Cohort A, which consists of patients with central nervous system symptoms.」
For JR-141's global Phase III trial, the company indicated plans to complete enrollment by the end of the first quarter of fiscal 2024 for the number of patients required for the interim analysis in Cohort A. For JR-441, completion of patient enrollment is expected in the second half of 2024, while the company continues to target the launch of a clinical trial for JR-446 during fiscal 2024. - FY2024-Q3AdvancedAnnual report
「For the blood-brain barrier-penetrating enzyme replacement therapy for mucopolysaccharidosis type IIIA (development code: JR-441), the CTA (clinical trial application) for the Phase I/II trial was accepted by Germany's regulatory authority, the Paul-Ehrlich Institute (PEI). Dosing of the first subject began in October 2023, and enrollment is currently under way. In December 2023, the product received orphan-drug designation from the US FDA. The Phase II clinical trial of the long-acting recombinant human growth hormone product (development code: JR-142) has been completed. An extension study is currently under way in patients who received treatment, and preparations are also under way to launch a Phase III clinical trial in fiscal 2024.」
JR-441 received orphan-drug designation from the US FDA in December 2023 while initial dosing and patient enrollment were under way in its Phase I/II trial. JR-142 completed its Phase II trial and progressed to an extension study and preparations to launch the Phase III trial in fiscal 2024. - FY2024-Q2AdvancedAnnual report
「For lepunafusp alfa, a blood-brain barrier-penetrating enzyme replacement therapy for mucopolysaccharidosis type I (development code: JR-171), the 13-week Phase I/II clinical trial in Japan, Brazil and the US has been completed, and an extension study is under way. Preparations are under way for the early launch of a global Phase III clinical trial. For the blood-brain barrier-penetrating enzyme replacement therapy for mucopolysaccharidosis type IIIA (development code: JR-441), the CTA (clinical trial application) for the Phase I/II trial was accepted by Germany's regulatory authority, the Paul-Ehrlich Institute (PEI). Dosing of the first subject began in October 2023, and enrollment is currently under way. For the blood-brain barrier-penetrating enzyme replacement therapy for mucopolysaccharidosis type IIIB (development code: JR-446), preparations are currently under way to launch a global clinical trial during fiscal 2024. The Phase II clinical trial of the long-acting recombinant human growth hormone product (development code: JR-142) has been completed. An extension study is currently under way in patients who received treatment, and preparations are also under way to launch a Phase III clinical trial.」
JR-171 completed its 13-week Phase I/II trial and progressed to an extension study; JR-441 progressed to initial dosing in its Phase I/II trial; and JR-142 completed its Phase II trial and progressed to an extension study and preparations for the Phase III trial. The target of launching a global clinical trial for JR-446 during fiscal 2024 was announced. - FY2024-Q1AdvancedAnnual report
「For the blood-brain barrier-penetrating enzyme replacement therapy for mucopolysaccharidosis type IIIA (development code: JR-441), the CTA (clinical trial application) for the Phase I/II trial was accepted by Germany's regulatory authority, the Paul-Ehrlich Institute (PEI). Final adjustments to the clinical trial protocol are currently under way, with the aim of starting recruitment in the first half of fiscal 2023.」
The CTA for JR-441's Phase I/II trial was accepted in Germany, and the program progressed to the final adjustment stage ahead of the planned start of recruitment in the first half of fiscal 2023. JR-171 completed enrollment in its Phase I/II trial and is undergoing final analysis, while JR-142 completed the statistical analysis for its Phase II trial and is being prepared for Phase III. Concrete development milestones progressed for multiple products. - FY2024-Q1AchievedAnnual report
「In June 2023, partial-change approval was obtained for the addition of short stature associated with SHOX abnormalities without epiphyseal closure as an indication for Growject® (development code: JR-401X).」
JR-401X progressed from the conventional stage of applying for marketing approval to obtaining partial-change approval, achieving the approval milestone for the added indication. - FY2023AdvancedAnnual report
「For lepunafusp alfa, a blood-brain barrier-penetrating enzyme replacement therapy for mucopolysaccharidosis type I (development code: JR-171), enrollment of all planned patients in the Phase I/II clinical trial in Japan, Brazil and the US was completed in March 2022, and final analysis is under way. Preparations are under way for the early launch of a global Phase III clinical trial. For the blood-brain barrier-penetrating enzyme replacement therapy for mucopolysaccharidosis type IIIA (development code: JR-441), preparations are currently under way to launch a global clinical trial in early fiscal 2023. A Phase II clinical trial of the recombinant long-acting growth hormone product (development code: JR-142) is under way; the planned statistical analysis has been completed, and preparations are under way to launch a Phase III clinical trial.」
JR-171 progressed from completion of enrollment and final analysis in the Phase I/II trial to preparations for the Phase III trial. The company is targeting the launch of global clinical trials for JR-441 in early fiscal 2023 and for JR-446 during fiscal 2024. JR-142 completed the statistical analysis for its Phase II trial and progressed to preparations for the Phase III trial. Enrollment is continuing in the global Phase III trial of JR-141, with resubmission in Brazil planned following the non-approval of the initial application. - FY2023-Q3AdvancedAnnual report
「In the United States, the FDA granted Fast Track designation in February 2021 and Rare Pediatric Disease designation in December 2022. In the European Union, the EMA granted PRIME designation in October 2021.」
JR-141 newly received Rare Pediatric Disease designation from the FDA in December 2022. The company continued to report the development status of the global Phase III trial, plans to refile in Brazil using the trial results after the previous application was rejected, and completion of enrollment of all subjects in JR-171's Phase I/II trial, which is undergoing final analysis. - FY2023-Q2AdvancedAnnual report
「For the blood-brain barrier-penetrating enzyme therapy for mucopolysaccharidosis type I, lepunafusp alfa (development code: JR-171), the company completed enrollment of all planned subjects in the Phase I/II clinical trial in Japan, Brazil and the United States in March 2022 and is conducting the final analysis.」
JR-171 completed enrollment of all planned subjects in its Phase I/II trial in Japan, Brazil and the United States and advanced to the final analysis stage. JR-142 also completed enrollment of its planned subjects in a Phase II trial. - FY2023-Q2WorsenedAnnual report
「The company had filed for marketing approval with ANVISA in Brazil in December 2020, but the application was rejected in August 2022. It plans to refile using the results of the ongoing global Phase III clinical trial.」
JR-141's marketing approval application in Brazil was rejected in August 2022, and the disclosure changed to state that the company plans to refile using the results of the global Phase III trial. - FY2023-Q1AdvancedAnnual report
「For the additional indication of Growject® in SHOX deficiency (development code: JR-401X), the company filed for marketing approval in July 2022.」
The additional indication of Growject® for SHOX deficiency advanced from the clinical trial stage to the marketing approval filing stage. - FY2022AdvancedAnnual report
「In February 2022, dosing of the first subject began in the global Phase III clinical trial.」
For JR-141, dosing of the first subject began in the global Phase III trial. JR-171 completed enrollment of all planned subjects by March 2022, and development of JR-479 was approved to begin in March 2022. - FY2022-Q3AdvancedAnnual report
「For the blood-brain barrier-penetrating enzyme therapy for mucopolysaccharidosis type IIIA (development code: JR-441), the EC granted orphan drug designation in January 2022, making the product eligible for various incentives to accelerate development in the European Union. Preparations are currently under way to begin global clinical trials in the first half of 2023.」
JR-441 received orphan drug designation from the EC and advanced to the preparation stage for the start of global clinical trials in the first half of 2023. - FY2022-Q2AdvancedAnnual report
「For the blood-brain barrier-penetrating enzyme therapy for mucopolysaccharidosis type I (development code: JR-171), Phase I/II clinical trials are currently being conducted in Japan, Brazil and the United States. The FDA granted orphan drug designation in February 2021 and the European Commission (EC) in March 2021. The FDA also granted Fast Track designation in September 2021, which is expected to accelerate clinical development in the United States and facilitate priority review and accelerated approval.」
JR-171 is currently undergoing Phase I/II trials in Japan, Brazil and the United States, and newly received FDA Fast Track designation. - FY2022-Q2AdvancedAnnual report
「For the blood-brain barrier-penetrating Hunter syndrome treatment pabinafusp alfa (development code: JR-141), the company began sales in Japan in May 2021 under the product name Izcargo® for IV infusion 10 mg. In Brazil, it filed for marketing approval with ANVISA in December 2020. In other regions, the FDA newly granted Fast Track designation in February 2021 and the EMA granted PRIME designation in October 2021. Preparations are currently under way to begin global Phase III clinical trials in the United States, Brazil and Europe.」
JR-141 began sales in Japan and, in addition to FDA Fast Track designation, received PRIME designation from the EMA, advancing preparations to begin global Phase III trials in the United States, Brazil and Europe. JR-171's Phase I/II trial was conducted in Japan, Brazil and the United States, and the product also received FDA Fast Track designation. - FY2022-Q1AdvancedAnnual report
「For the blood-brain barrier-penetrating Hunter syndrome treatment pabinafusp alfa (development code: JR-141; product name: Izcargo® for IV infusion 10 mg), the company began sales in Japan in May 2021. In Brazil, it filed for marketing approval with ANVISA in December 2020. In other regions, the FDA granted orphan drug designation in October 2018 and the EMA in February 2019, while in February 2021 the FDA granted Fast Track designation and approved the start of a Phase III clinical trial. Preparations are under way to begin the global clinical trial in Brazil and Europe following the United States. For the blood-brain barrier-penetrating enzyme therapy for mucopolysaccharidosis type I (development code: JR-171), the company has been conducting a Phase I/II global clinical trial in Japan and Brazil since October 2020, and trial initiation has also been approved in the United States. The FDA granted orphan drug designation in February 2021 and the EMA in March 2021.」
JR-141 reached the commercial launch stage in Japan and advanced preparations to begin global Phase III trials in Brazil and Europe following the United States. JR-171's Phase I/II trial was conducted in Japan and Brazil, with approval to begin the trial in the United States and orphan drug designations from the FDA and EMA. - FY2021AdvancedAnnual report
「For the blood-brain barrier-penetrating Hunter syndrome treatment pabinafusp alfa (development code: JR-141; Izcargo® for IV infusion 10 mg), the company obtained marketing approval in Japan in March 2021. In Brazil, it filed for marketing approval with ANVISA in December 2020. In other regions, the FDA granted orphan drug designation in October 2018 and the EMA in February 2019, and in February 2021 the FDA designated the product for Fast Track and approved the start of a Phase III clinical trial.」
JR-141 obtained marketing approval in Japan and advanced to a marketing approval filing in Brazil, FDA Fast Track designation and approval to begin a global Phase III clinical trial. For JR-171, global clinical development progressed after dosing of the first subject. JR-142 also began its Phase II clinical trial. - FY2021-Q3AdvancedAnnual report
「For the blood-brain barrier-penetrating Hunter syndrome treatment pabinafusp alfa (development code: JR-141), the company filed for marketing approval in Japan in September 2020. JR-141 was designated by the Ministry of Health, Labour and Welfare in March 2018 under the Sakigake designation system. In Brazil, the company filed for marketing approval with ANVISA in December 2020, while in other regions the FDA granted orphan drug designation in October 2018 and the EMA in February 2019. The company is preparing for global development, including discussions with the FDA on starting a global Phase III clinical trial. For the blood-brain barrier-penetrating enzyme therapy for mucopolysaccharidosis type I (development code: JR-171), the company's first global clinical trial, the Phase I/II clinical trial notification was accepted by the PMDA in Japan in July 2020, and the first subject received the investigational drug in October 2020. Trial initiation was also approved by ANVISA and the FDA. In December 2020, the company submitted a clinical trial notification to the PMDA for a Phase II trial of the recombinant long-acting growth hormone preparation JR-142, scheduled to begin in March 2021.」
JR-141 advanced to a marketing approval filing in Brazil following Japan, and the company began discussions with the FDA on starting a global Phase III trial. For JR-171, the first subject was dosed in Japan, and trial initiation was approved by ANVISA and the FDA. JR-142 reached the clinical trial notification stage for its Phase II trial, scheduled to begin in March 2021. - FY2021-Q2AdvancedAnnual report
「For the blood-brain barrier-penetrating enzyme therapy for mucopolysaccharidosis type I (development code: JR-171), the company submitted and received acceptance of clinical trial notifications for a Phase I/II trial to the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in July this year and to Brazil's National Health Surveillance Agency (ANVISA) in October this year. This is the company's first global clinical trial, and the trial will begin in Japan ahead of Brazil and the United States.」
Clinical trial notifications for the Phase I/II trial of JR-171 were accepted in Japan and Brazil, advancing the program to the stage of beginning the company's first global clinical trial in Japan. - FY2021-Q2AdvancedAnnual report
「For the blood-brain barrier-penetrating Hunter syndrome treatment pabinafusp alfa (development code: JR-141), the company filed for marketing approval in Japan in September 2020.」
JR-141 advanced to the marketing approval filing stage in Japan. - FY2021-Q1RestatedAnnual report
「For the blood-brain barrier-penetrating Hunter syndrome treatment JR-141 (development code: JR-141), the company began a Phase II clinical trial in Brazil in June 2018 and a Phase III clinical trial in Japan in August 2018. In other regions, the FDA granted orphan drug designation in October 2018 and the EMA granted orphan drug designation in February 2019, while the company continued preparing for global development.」
The company reiterated JR-141's Phase II trial in Brazil, Phase III trial in Japan, orphan drug designations from the FDA and EMA, and preparations for global development. R&D is also being conducted sequentially on other J-Brain Cargo®-based products, including JR-162, JR-171, JR-441, JR-443 and JR-446, but no new trial phase or marketing approval filing was reported. - FY2020AdvancedAnnual report
「In fiscal 2020, the company plans to file for approval of JR-141 in Japan and Brazil and to begin global clinical trials for JR-141 and the Hurler syndrome treatment JR-171.」
For JR-141, the company planned approval filings in Japan and Brazil during fiscal 2020 and the start of global clinical trials including JR-171. It had advanced to the stage of targeting the start of global clinical trials for multiple products, including JR-162 and JR-441, over the following 3 years. - FY2020-Q3RestatedAnnual report
「JR-141's Phase II clinical trial in Brazil began in June the year before last, and its Phase III clinical trial in Japan began in August of that year. Both clinical trials progressed smoothly. In other regions, the FDA granted orphan drug designation in October the year before last and the European Medicines Agency (EMA) granted orphan drug designation in February last year, as the company steadily prepared for global development. Subsequently, the company sequentially began R&D on lysosomal disease treatments including the Pompe disease enzyme therapy JR-162, the Hurler syndrome enzyme therapy JR-171 and the type A Sanfilippo syndrome enzyme therapy JR-441, and will pursue global development of these new drugs.」
The company reconfirmed JR-141's Phase II trial in Brazil, Phase III trial in Japan, and orphan drug designations from the FDA and EMA, as well as R&D on JR-162, JR-171 and JR-441. No explicit milestones such as a transition to a new trial phase or a marketing approval filing were reported. - FY2020-Q2SoftenedAnnual report
「The company temporarily withdrew its marketing approval application in September this year for the expanded indication of subcutaneous Temcell® HS Inj. in epidermolysis bullosa, having determined that it was necessary to demonstrate the product's efficacy for this indication more clearly.」
JR-031EB's marketing approval application was temporarily withdrawn, and the company is considering its future development policy. - FY2020-Q2AdvancedAnnual report
「In the cell and regenerative medicine field, the company entered into a joint development agreement and license agreement with Teijin Limited in July the year before last for JTR-161/JR-161, a regenerative medicine product using allogeneic dental pulp-derived stem cells for acute ischemic stroke, and began administering the product to patients in a Phase I/II clinical trial in February this year. In addition, it began a Phase I/II clinical trial in July this year for a new indication of Temcell® HS Inj. in neonatal hypoxic-ischemic encephalopathy (development code: JR-031HIE).」
JTR-161/JR-161 began administration to patients in a Phase I/II clinical trial, and the Phase I/II clinical trial for the expanded JR-031HIE indication of Temcell® HS Inj. also began. - FY2020-Q2AchievedAnnual report
「On 20 September, the Company obtained marketing approval for the long-acting erythropoiesis-stimulating agent darbepoetin alfa BS injection 「JCR」, jointly developed with キッセイ薬品工業株式会社.」
JR-131 advanced from the previous marketing approval filing stage to approval, achieving the approval milestone. - FY2020-Q1AdvancedAnnual report
「In addition, the company began a Phase I clinical trial in May this year for the recombinant long-acting growth hormone preparation JR-142.」
The company began a Phase I clinical trial for the long-acting growth hormone preparation JR-142, advancing the development pipeline from the preclinical to the clinical development stage. - FY2019AdvancedAnnual report
「The company filed for marketing approval of the darbepoetin alfa biosimilar JR-131 last September and for an expanded indication of the regenerative medicine product Temcell® HS Inj. for epidermolysis bullosa (development code: JR-031EB) in March this year. It also began Phase II clinical trials in Brazil and Phase III clinical trials in Japan for the Hunter syndrome enzyme therapy JR-141, as well as a clinical trial for JTR-161, a regenerative medicine product using human dental pulp-derived stem cells for acute ischemic stroke.」
JR-131 and JR-031EB advanced to the marketing approval filing stage. JR-141 began its Phase II trial in Brazil and Phase III trial in Japan, while JTR-161 also reached the clinical trial start stage. - FY2019-Q3AdvancedAnnual report
「JR-141's Phase II clinical trial in Brazil began in June last year, its Phase III clinical trial in Japan began in August last year and patient enrollment was completed in December. In addition, the US Food and Drug Administration (FDA) granted orphan drug designation last October, and development is progressing smoothly. Subsequently, the company began developing the enzyme therapy for type A Sanfilippo syndrome JR-441 last September as a new drug incorporating J-Brain Cargo®. It is sequentially conducting R&D on 15 lysosomal disease treatments, including the Pompe disease enzyme therapy JR-162 and the Hurler syndrome enzyme therapy JR-171, and plans to pursue global development for all of them.」
JR-141 completed patient enrollment in its Phase III clinical trial in Japan in December 2018 and received orphan drug designation from the FDA. Development of JR-441 also began, expanding the development portfolio to 15 products, including JR-162 and JR-171. - FY2019-Q2AdvancedAnnual report
「In R&D, the company began a Phase III clinical trial in July this year for an additional indication of the growth hormone preparation Growject® in patients with SHOX deficiency. On 28 September this year, it filed for marketing approval of the darbepoetin alfa biosimilar JR-131, being jointly developed with Kissei Pharmaceutical Co., Ltd. In its priority lysosomal disease drug development program, the company obtained marketing approval on 21 September this year for the Fabry disease enzyme therapy agalsidase beta BS for IV infusion "JCR" (development code: JR-051), the first domestically produced lysosomal disease enzyme therapy in Japan. The company is currently developing innovative new drugs using its proprietary blood-brain barrier transport technology J-Brain Cargo®. It began a Phase II clinical trial in Brazil in June this year for the Hunter syndrome enzyme therapy JR-141, and began its Phase III clinical trial in Japan in August.」
JR-131 entered the marketing approval filing stage, while JR-051 received approval on 21 September. Following its Phase II trial in Brazil, JR-141 began its Phase III trial in Japan, and the Phase III trial for SHOX deficiency also began. - FY2019-Q1AdvancedAnnual report
「In R&D, the company began a Phase III clinical trial in July this year in patients with SHOX deficiency to add an indication for Growject®, while the long-acting growth hormone preparation JR-142 remains in preclinical development. In the lysosomal disease field, the company filed for marketing approval of the agalsidase beta biosimilar for Fabry disease (development code: JR-051) last September. It also began a Phase II clinical trial in Brazil in June this year for the Hunter syndrome enzyme therapy JR-141 incorporating J-Brain Cargo®, with a Phase III clinical trial in Japan scheduled to begin in August. Going forward, the company plans to advance development of the J-Brain Cargo®-based Pompe disease enzyme therapy JR-162 and the Hurler syndrome enzyme therapy JR-171, and aims to add these and 12 further lysosomal diseases and their central nervous system disorders to its development pipeline as soon as possible after basic research.」
The company began the Phase III trial of Growject® for SHOX deficiency, started the Phase II trial of JR-141 in Brazil and scheduled the start of its Phase III trial in Japan for August. JR-051 had been filed for approval, JR-131 was being prepared for filing, and the company also stated its policy of expanding the lysosomal disease development targets by 12 additional indications. - FY2018AdvancedAnnual report
「JR-141 is an innovative Hunter syndrome enzyme therapy using the blood-brain barrier transport technology J-Brain Cargo®, expected to treat central nervous system symptoms for which existing products have shown no therapeutic effect. In March 2018, it was designated under Japan's Sakigake designation system. In the Phase I/II trial launched last March, no safety issues were identified and results consistent with expectations were obtained, suggesting that the drug crossed the blood-brain barrier and acted on the central nervous system. The company will give top priority to development toward starting a Phase III trial in 2018 and filing for marketing approval in Japan in 2019.」
JR-141 received Sakigake designation and produced safe, favorable results in its Phase I/II trial. The company presented plans to start a Phase III trial and file for approval in Japan in 2019. For JR-131, the company is evaluating equivalence to the reference product in a Phase III trial, targeting an approval filing during 2018. - FY2018-Q3RestatedAnnual report
「In R&D, the company filed for marketing approval of the agalsidase beta biosimilar for Fabry disease (development code: JR-051) last September. It also progressed smoothly as planned with the Phase III clinical trial for the darbepoetin alfa biosimilar JR-131 and the Phase I/II clinical trial for the Hunter syndrome enzyme therapy JR-141 incorporating J-Brain Cargo®, with favorable results obtained in both clinical development programs. Preclinical candidates, including the long-acting growth hormone preparation JR-142, the J-Brain Cargo®-based Pompe disease enzyme therapy JR-162 and the Hurler syndrome enzyme therapy JR-171, also continued to progress smoothly.」
The company reconfirmed the filing for JR-051, the Phase III trial for JR-131, the Phase I/II trial for JR-141 and progress on preclinical candidates. Although favorable results were reported in clinical development, no new approval or transition to a new trial phase was reported. - FY2018-Q2AdvancedAnnual report
「On 29 September of this year, the company filed for marketing approval of the agalsidase beta biosimilar for Fabry disease (development code: JR-051), while progressing smoothly as planned with the Phase III clinical trial for the darbepoetin biosimilar JR-131 and the Phase I/II clinical trial for the Hunter syndrome enzyme therapy JR-141 incorporating J-Brain Cargo®.」
JR-051 entered the marketing approval filing stage, following submission of the application and advancing toward the approval milestone. JR-131's Phase III trial and JR-141's Phase I/II trial progressed as planned. - FY2018-Q1AdvancedAnnual report
「In R&D, the Phase III clinical trial for the darbepoetin biosimilar JR-131 and the Phase I/II clinical trial for the Hunter syndrome enzyme therapy JR-141, incorporating the company's proprietary blood-brain barrier transport technology J-Brain Cargo®, are progressing smoothly as planned. For the agalsidase beta biosimilar JR-051, which is in Phase II/III clinical trials, the company advanced preparations for a marketing approval filing targeted for this summer. Preclinical R&D also progressed on the long-acting growth hormone preparation JR-142 and the Pompe disease enzyme therapy JR-162 incorporating J-Brain Cargo®, among other projects.」
The company advanced JR-131's Phase III trial and JR-141's Phase I/II trial as planned, while JR-051 progressed to the preparation stage for a marketing approval filing targeted for this summer. It also reported progress in preclinical research on JR-142, JR-162 and other projects. - FY2017AdvancedAnnual report
「The company completed patient enrollment in the final clinical Phase II/III trial in October last year and is preparing supply, sales and other systems for rapid market penetration, with a view to filing for marketing approval in fiscal 2017 and obtaining approval during fiscal 2018. The Phase III clinical trial launched last August is progressing smoothly, and development will continue steadily toward a marketing approval filing during fiscal 2018. In March this year, the company began a Phase I/II clinical trial in patients with Hunter syndrome.」
JR-051 completed patient enrollment in its Phase II/III trial, with a plan to file in fiscal 2017 and obtain approval in fiscal 2018. JR-131's Phase III trial was progressing, and JR-141's Phase I/II trial began in March 2017. - FY2017-Q3AdvancedAnnual report
「In R&D, the biosimilar of the long-acting erythropoiesis-stimulating agent darbepoetin alfa (development code: JR-131), which is in Phase III clinical trials, and the biosimilar of the Fabry disease treatment enzyme agalsidase beta (development code: JR-051), which is in Phase II/III clinical trials, are progressing smoothly as planned.」
The company reconfirmed that JR-131's Phase III and JR-051's Phase II/III clinical trials were progressing as planned. JR-141 had also entered the final preparation stage for the start of clinical trials during the current fiscal year. - FY2017-Q2AdvancedAnnual report
「In R&D, the biosimilar of the long-acting erythropoiesis-stimulating agent darbepoetin alfa (development code: JR-131) has advanced to Phase III clinical trials, while Phase II/III clinical trials for the biosimilar of the Fabry disease treatment enzyme agalsidase beta (development code: JR-051) are also progressing smoothly.」
The company stated that JR-131 had advanced to Phase III clinical trials and that JR-051 was progressing through Phase II/III clinical trials. - FY2017-Q1AdvancedAnnual report
「In R&D, clinical trials for biosimilars of the Fabry disease treatment enzyme agalsidase beta (development code: JR-051) and the long-acting erythropoiesis-stimulating agent darbepoetin alfa (development code: JR-131) are progressing smoothly. Research and development on various other projects, including the long-acting growth hormone preparation JR-142, which is expected to further improve patients' quality of life, is also progressing smoothly.」
The company reported progress in clinical trials for JR-051 and JR-131 and stated that R&D for JR-142 and other projects was also progressing smoothly. It did not report achievement of approval or filing milestones. - FY2016First statedAnnual report
「For the development pipeline, the company plans to obtain approval by fiscal 2019 for biosimilars of agalsidase beta (development code: JR-051) and darbepoetin alfa (development code: JR-131), and to file for approval of a blood-brain barrier-penetrating treatment for Hunter syndrome (development code: JR-141).」
Specific product-level milestones through fiscal 2019 include plans to obtain approval for 2 products and file for approval for 1 product.