JR-441 / JCR Pharma - LARVOL DELTA

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA (clinicaltrials.gov) - P1/2 | N=15 | Active, not recruiting | Sponsor: JCR Pharmaceuticals Co., Ltd. | Recruiting ➔ Active, not recruiting

Enrollment closed • Hunter Syndrome • Lysosomal Storage Diseases

U.S. FDA Grants Orphan Drug Designation to JR-441 for the Treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA) (Businesswire) - "JCR Pharmaceuticals Co., Ltd...announced that the U.S. Food and Drug Administration ('FDA') granted orphan drug designation (ODD) to JR-441, an investigational drug for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, or Sanfilippo syndrome type A). JR-441 is a blood-brain barrier (BBB)-penetrating form of recombinant heparan N-sulfatase that was developed using JCR’s proprietary J-Brain Cargo BBB-penetrating technology....JCR is currently conducting a global Phase I/II clinical trial for JR-441, with the first patient with MPS IIIA dosed in October 2023."

Orphan drug • Trial status • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: JCR Pharmaceuticals Co., Ltd.

New P1/2 trial • Hunter Syndrome • Lysosomal Storage Diseases