treatment of Pitt Hopkins syndrome
All entries for: Orphan Drug Designation
March 8, 2026
OcuNeo Therapeutics Inc.
Orphan Drug Designation
Sioux Falls
1-50 employees
treatment of vernal keratoconjunctivitis
Disease Area: Rare Disease
March 6, 2026
GENFIT
Orphan Drug Designation
treatment of acute-on-chronic liver failure
Disease Area: Rare Disease
March 6, 2026
Aminex Therapeutics, Inc.
Orphan Drug Designation
Kenmore
1-50 employees
prevention of allograft rejection in liver transplantation
Disease Area: Rare Disease
March 5, 2026
Aminex Therapeutics, Inc.
Orphan Drug Designation
Kenmore
1-50 employees
treatment of malignant glioma
Disease Area: Rare Disease
March 5, 2026
Multitude Therapeutics Inc.
Orphan Drug Designation
Shanghai
1-50 employees
treatment of malignant glioma
Disease Area: Rare Disease
March 5, 2026
Multitude Therapeutics Inc.
Orphan Drug Designation
Shanghai
1-50 employees
treatment of soft tissue sarcoma
Disease Area: Rare Disease
March 5, 2026
RNR Biomedical inc
Orphan Drug Designation
Tenafly
1-50 employees
treatment of Behcet disease
Disease Area: Rare Disease
March 2, 2026
Shanghai Allovanta Biotechnology Co., Ltd.
Orphan Drug Designation
Shanghai
1-50 employees
treatment of follicular lymphoma
Disease Area: Rare Disease
March 2, 2026
Takeda Development Center Americas, Inc.
Orphan Drug Designation
Cambridge
1-50 employees
treatment of narcolepsy type 1
Disease Area: Rare Disease