Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by the U.S. FDA

Business Wire

07-05-2025

TOKYO & HYOGO, Japan--(BUSINESS WIRE)-- MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B).
MPS IIIB affects an estimated 500 to 1,000 individuals worldwide, 1 causing severe central nervous system (CNS) symptoms. Despite the dire need, there are currently no approved treatments available for this condition. JR-446, developed using JCR's proprietary J-Brain Cargo ® technology, has shown promising non-clinical results in addressing the CNS symptoms of this challenging disorder, and is currently being studied in a Phase I/II trial that is being conducted in Japan (JR- 446-101) under a collaboration agreement between the two companies.
'We are extremely pleased that JR-446 has received orphan drug designation from the U.S. FDA,' said Shuichi Watanabe, Representative Director, President and CEO of MEDIPAL. 'We will continue the development of JR-446 in collaboration with JCR so that we can soon deliver this drug to the patients and their families who are desperately waiting, as there is currently no treatment available.'
'This orphan drug designation is an important development step for the JR-446 clinical program,' said Shin Ashida, President, Chairman and CEO of JCR Pharmaceuticals. 'This orphan drug designation supports the potential of this therapy to address the significant unmet medical need of patients with MPS IIIB.'
In September 2023, MEDIPAL and JCR entered into a licensing agreement in which MEDIPAL will commercialize JR-446 outside of Japan. In addition, MEDIPAL will support JCR in the clinical development of JR-446 in Japan, including the distribution of investigational drugs, disease awareness, and clinical trial advancement. 2
With the ODD, JR-446 will be eligible for various incentives to encourage the development in the U.S.
About Orphan Drug Designation in the U.S.
The U.S. FDA's Office of Orphan Products Development grants orphan status to drugs being developed to treat, prevent, or diagnose a rare disease or condition affecting fewer than 200,000 people in the U.S. The designation provides significant incentives to promote the development of the drug including the potential for market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, and waiver of Prescription Drug User Fee Act Application fee.
About Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B) Mucopolysaccharidosis type IIIB, or Sanfilippo syndrome type B, is an autosomal recessive disease caused by pathogenic mutations in the NAGLU gene, encoding a lysosomal enzyme involved in the degradation of heparan sulfate. With the accumulation of heparan sulfate in the central nervous system in the brain, individuals with this condition present rapid neurological decline, including sleep disorders, loss of speech, and behavioral changes, which may significantly affect the quality of life of patients and their families.
About the J-Brain Cargo ® Platform Technology
JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology, J- Brain Cargo ®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology is IZCARGO ® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder.
About MEDIPAL HOLDINGS CORPORATION
MEDIPAL is a holding company which controls, administers and supports the operating activities of companies in which it holds shares in the Prescription Pharmaceutical Wholesale Business; the Cosmetics, Daily Necessities and OTC Pharmaceutical Wholesale Business; and the Animal Health Products and Food Processing Raw Materials Wholesale and Related Business, and conducts business development for the MEDIPAL Group. For more information, visit https://www.medipal.co.jp/english/.
About JCR Pharmaceuticals Co., Ltd.
JCR is a global specialty pharmaceuticals company dedicated to advancing treatments for rare and genetic diseases. With nearly 50 years of expertise in Japan, JCR is expanding to the US, Europe, and Latin America. JCR's innovative therapies address conditions like growth disorder, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. JCR is also developing treatments for rare diseases like MPS I, MPS II, MPS IIIA and B, and more. The core values of reliability, confidence, and persistence drive JCR's mission to enhance global medical progress. For more information, visit https://www.jcrpharm.co.jp/en/site/en/.
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References
Based on data from JCR's own investigations, referring to the Ministry of Health, Labour and Welfare's public research.
Reference: Press release on the licensing agreement for JR-446 between MEDIPAL and JCR (September 28, 2023).