ASHIYA, Japan—JCR Pharmaceuticals Co., Ltd. has reported that the European Medicines Agency has granted orphan drug designation to JR-171, JCR’s investigational drug for the treatment of mucopolysaccharidosis type I (MPS I) — also known as Hurler, Hurler-Scheie, and Scheie syndrome, dependent upon severity. 

MPS I is a lysosomal storage disorder caused by a deficiency of α-L-iduronidase, an enzyme that breaks down glycosaminoglycans (mucopolysaccharides) in the body. MPS I gives rise to a wide range of somatic and neurological symptoms, and current enzyme replacement therapies do not address those which affect the CNS, since they can’t cross the blood-brain barrier (BBB).

JR-171 is a BBB-penetrating form recombinant α-L-iduronidase developed using JCR’s J-Brain Cargo. JCR’s proprietary J-Brain Cargo technology reportedly enables the development of therapies that can cross the BBB and penetrate the CNS. The CNS complications of lysosomal storage disorders can be severe, resulting in developmental delays, an impact on cognition, and poor prognosis, which affects patients’ independence and quality of life. JCR wants to address the clinical challenges of lysosomal storage disorders by delivering the enzyme to both body and brain.

JR-171 previously received Orphan Drug designation from the FDA in February. The therapy is currently in a global Phase 1/2 clinical trial, and the first patient in Japan was dosed in October 2020. The trial is also underway in Brazil, and dosed the first patient this March. This trial will also enroll patients in the US.

JCR also announced yesterday that results of the Phase 2 clinical trial in Brazil of the company’s JR-141 (pabinafusp alfa) therapy for the treatment of mucopolysaccharidosis II (MPS II, also known as Hunter syndrome) have been published in Molecular Therapy. JR-141 is a BBB-penetrating recombinant iduronate-2-sulfatase product for the treatment of patients with MPS II, which was also created with JCR’S J-Brain Cargo technology. 

JR-141 recently received Marketing Approval in Japan, and JCR also filed an application in December 2020 to the Agência Nacional de Vigilância Sanitária in Brazil for marketing authorization of JR-141 for the treatment of MPS II. The company has also noted that preparations are underway for a global Phase 3 clinical trial for JR-141 in the US, Brazil, and Europe. The FDA recently accepted an IND application for JR-141 to begin the planned clinical study.