JR-446 / JCR Pharma, MEDIPAL HOLDINGS CORPORATION - LARVOL DELTA

Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by Ministry of Health, Labour and Welfare of Japan (Businesswire)

Orphan drug • Lysosomal Storage Diseases

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB (clinicaltrials.gov) - P1/2 | N=10 | Recruiting | Sponsor: JCR Pharmaceuticals Co., Ltd. | Trial completion date: Dec 2028 ➔ Apr 2030 | Trial primary completion date: Jun 2026 ➔ Apr 2030

Trial completion date • Trial primary completion date • Hunter Syndrome • Lysosomal Storage Diseases

Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by European Commission (EC) (Businesswire) - "MEDIPAL HOLDINGS CORPORATION...and JCR Pharmaceuticals Co., Ltd...today announced that the European Commission (EC) has 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)....With the ODD, JR-446 will be eligible for various incentives to encourage the development in the European Union (EU)."

Orphan drug • Lysosomal Storage Diseases

FDA grants orphan drug status to novel MPS IIIB treatment (Investing.com) - "MEDIPAL HOLDINGS CORPORATION (TSE:7459) and JCR Pharmaceuticals Co., Ltd. (TSE:4552) have announced the U.S. Food and Drug Administration’s orphan drug designation for their investigational drug JR-446, aimed at treating mucopolysaccharidosis type IIIB (MPS IIIB), also known as Sanfilippo syndrome type B."

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

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB (clinicaltrials.gov) - P1/2 | N=10 | Recruiting | Sponsor: JCR Pharmaceuticals Co., Ltd. | Not yet recruiting ➔ Recruiting | Trial completion date: Sep 2029 ➔ Dec 2028 | Trial primary completion date: Mar 2027 ➔ Jun 2026

Enrollment open • Trial completion date • Trial primary completion date • Hunter Syndrome • Lysosomal Storage Diseases

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB (clinicaltrials.gov) - P1/2 | N=10 | Not yet recruiting | Sponsor: JCR Pharmaceuticals Co., Ltd.

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