prevention of placenta-mediated adverse pregnancy outcomes in pregnant patients with antiphospholipid syndrome
All entries for: Rare Disease
March 2, 2026
UCB, Inc.
Orphan Drug Designation
Smyrna
1-50 employees
Disease Area: Rare Disease
March 2, 2026
AniMusCure Inc.
Orphan Drug Designation
treatment of amyotrophic lateral sclerosis
Disease Area: Rare Disease
March 2, 2026
AbbVie Inc.
Orphan Drug Designation
treatment of neuroendocrine carcinoma
Disease Area: Rare Disease
March 2, 2026
Kopra Bio, Inc.
Orphan Drug Designation
San Francisco,
1-50 employees
treatment of malignant glioma
Disease Area: Rare Disease
March 2, 2026
PMV Pharmaceuticals, Inc.
Orphan Drug Designation
Princeton
1-50 employees
treatment of TP53 Y220C positive ovarian cancer, fallopian tube cancer, and primary peritoneal cancer
Disease Area: Rare Disease
March 2, 2026
BlossomHill Therapeutics
Orphan Drug Designation
San Diego
1-50 employees
treatment of acute myeloid leukemia
Disease Area: Rare Disease
February 27, 2026
Evolyra Therapeutics
Orphan Drug Designation
treatment of limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5)
Disease Area: Rare Disease
February 27, 2026
CELLeBRAIN Co., Ltd.
Orphan Drug Designation
Jeonju-si
1-50 employees
treatment of malignant glioma
Disease Area: Rare Disease
February 27, 2026
Canwell Pharma, Inc.
Orphan Drug Designation
Wellesley
1-50 employees
treatment of soft tissue sarcoma
Disease Area: Rare Disease
February 27, 2026
Wenling City Innovation Biotechnology Co., Ltd.
Orphan Drug Designation
Wenling City
1-50 employees
treatment of amyotrophic lateral sclerosis
Disease Area: Rare Disease