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27 Jun 2025
Admin
News Article

JR-446 for mucopolysaccharidosis type IIIB recevies ODD from European Commission

Overview

Medipal Holdings Corporation and JCR Pharmaceuticals Co Ltd announced that the European Commission (EC) has granted orphan drug designation to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B), following the recent the US Food and Drug Administration designation.

About the medical condition: mucopolysaccharidosis type IIIB

MPS IIIB affects an estimated 500 to 1,000 individuals worldwide, 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 it is currently being studied in a phase I/II trial that is being conducted in Japan under a collaboration agreement between the two companies.

Medipal and JCR Partner to Advance JR-446 for MPS IIIB

In September 2023, Medipal Holdings Corporation and JCR Pharmaceuticals entered into a licensing agreement for JR-446, a therapeutic candidate for Mucopolysaccharidosis type IIIB (MPS IIIB).
Under the agreement, Medipal Holdings will commercialize JR-446 outside of Japan, expanding the therapy’s global reach.
In Japan, Medipal will support JCR’s clinical development efforts, including:
1. Distribution of investigational drugsRaising disease awareness
2. Advancing clinical trials
This collaboration aims to accelerate the development and global availability of JR-446 for patients with MPS IIIB.
With the ODD, JR-446 will be eligible for various incentives to encourage the development in the European Union.

ODD by European Commission

European Commission implements orphan designation drug for promoting new drug development for rare diseases in which the prevalence of the condition affects no more than five in 10,000 people in the European Union.
Designated drugs are granted market exclusivity for 10 years in the EU, as well as scientific guidance.
Fee reductions are also available depending on the status of the sponsor and the type of service required.

Mechanism behind the genetic diseases

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.

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