MEDIPAL HOLDINGS CORPORATION and JCR Pharmaceuticals Co., Ltd. announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to JR-446, an investigational therapy for mucopolysaccharidosis type IIIB (MPS IIIB), also known as Sanfilippo syndrome type B.
This rare genetic disorder affects an estimated 500 to 1,000 individuals worldwide and causes severe central nervous system (CNS) symptoms. Despite the significant medical need, there are currently no approved treatments available for MPS IIIB patients.
JR-446 was developed using JCR's proprietary J-Brain Cargo® technology, which enables biotherapeutics to penetrate the blood-brain barrier and reach the central nervous system. The investigational drug has demonstrated promising results in non-clinical studies specifically addressing the CNS symptoms characteristic of this disorder.
"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."
Shin Ashida, President, Chairman and CEO of JCR Pharmaceuticals, added, "This orphan drug designation is an important development step for the JR-446 clinical program. This orphan drug designation supports the potential of this therapy to address the significant unmet medical need of patients with MPS IIIB."
Current Clinical Development Status
JR-446 is currently being evaluated in a Phase I/II clinical trial (JR-446-101) in Japan under a collaboration agreement between MEDIPAL and JCR. The companies entered into a licensing agreement in September 2023, under which MEDIPAL will commercialize JR-446 outside of Japan while supporting JCR in the clinical development within Japan, including investigational drug distribution, disease awareness initiatives, and clinical trial advancement.
Understanding MPS IIIB
Mucopolysaccharidosis type IIIB is an autosomal recessive disease caused by mutations in the NAGLU gene, which encodes a lysosomal enzyme involved in the degradation of heparan sulfate. The condition leads to accumulation of heparan sulfate in the central nervous system, resulting in rapid neurological decline.
Patients typically experience sleep disorders, loss of speech, and behavioral changes that significantly impact quality of life for both patients and their families. The progressive nature of the disease and lack of approved treatments highlight the urgent need for therapeutic options.
Benefits of Orphan Drug Designation
The FDA's orphan drug designation provides several significant incentives to encourage the development of treatments for rare diseases affecting fewer than 200,000 people in the United States. These benefits include:
- Potential market exclusivity for seven years upon FDA approval
- Eligibility for tax credits for qualified clinical trials
- Waiver of Prescription Drug User Fee Act application fees
These incentives are designed to accelerate the development of treatments for rare conditions like MPS IIIB where the limited patient population might otherwise discourage pharmaceutical investment.
J-Brain Cargo® Technology Platform
The J-Brain Cargo® platform technology represents a significant advancement in addressing central nervous system disorders. This proprietary blood-brain barrier-penetrating technology enables the delivery of biotherapeutics directly to the central nervous system, overcoming one of the major challenges in treating neurological conditions.
The first drug developed using this technology, IZCARGO® (pabinafusp alfa), has already received approval in Japan for the treatment of a lysosomal storage disorder, demonstrating the platform's potential for addressing similar rare genetic conditions.
Company Collaboration
The partnership between MEDIPAL and JCR combines complementary expertise to advance JR-446 through clinical development. MEDIPAL brings extensive experience in pharmaceutical distribution and commercialization, while JCR contributes nearly five decades of expertise in developing treatments for rare and genetic diseases.
JCR Pharmaceuticals has established itself as a leader in rare disease therapeutics, with treatments addressing conditions including growth disorders, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. The company continues to expand its global presence beyond Japan into the US, Europe, and Latin America.
This orphan drug designation represents an important milestone in the companies' efforts to address the significant unmet need for patients suffering from MPS IIIB, potentially offering hope to patients and families affected by this devastating condition.
