treatment of gastric cancer, including gastroesophageal junction cancer
All entries for: Orphan Drug Designation
February 9, 2026
Hangzhou Zhongmei Huadong Pharmaceutical Co., Ltd
Orphan Drug Designation
Disease Area: Rare Disease
February 5, 2026
ENCell Co., Ltd.
Orphan Drug Designation
treatment of Duchenne muscular dystrophy
Disease Area: Rare Disease
February 5, 2026
Hellene Solomon Wan, MD - Induvidual sponsor.
Orphan Drug Designation
treatment of neonatal diabetes mellitus (monogenic forms of diabetes mellitus that present in infancy)
Disease Area: Rare Disease
February 4, 2026
Zydus Healthcare (USA), LLC,
Orphan Drug Designation
Treatment of Sickle Cell Disease
Disease Area: Rare Disease
February 4, 2026
Novartis Pharmaceuticals Corporation
Orphan Drug Designation
treatment of progressive supranuclear palsy
Disease Area: Rare Disease
February 3, 2026
Immatics US, Inc
Orphan Drug Designation
treatment of Stage II and higher cutaneous melanoma (CM), in human leukocyte antigen (HLA)-A*02:01-positive patients
Disease Area: Rare Disease
February 3, 2026
LBR Regulatory and Clinical Consulting Services, Inc.
Orphan Drug Designation
treatment of tuberous sclerosis complex
Disease Area: Rare Disease
February 2, 2026
ODDIFACT SAS
Orphan Drug Designation
treatment of neuromyelitis optica spectrum disorder
Disease Area: Rare Disease
February 2, 2026
Solve FSHD
Orphan Drug Designation
treatment of facioscapulohumeral muscular dystrophy
Disease Area: Rare Disease
February 2, 2026
Vertex Pharmaceuticals Incorporated
Orphan Drug Designation
treatment of myasthenia gravis
Disease Area: Rare Disease