Methodology

All entries for: Rare Disease

February 15, 2026

Elpiscience (Suzhou) Biopharma, Ltd.

Orphan Drug Designation

treatment of desmoid tumors

Disease Area: Rare Disease
Source
Source
February 14, 2026

InGel Therapeutics Inc.

Orphan Drug Designation

treatment of retinitis pigmentosa

Disease Area: Rare Disease
Source
Source
February 13, 2026

Zomagen Biosciences Ltd, a wholly owned subsidiary of Ventyx Biosciences, Ltd.

Orphan Drug Designation

treatment of pericarditis

Disease Area: Rare Disease
Source
Source
February 13, 2026

Bristol Myers Squibb

Orphan Drug Designation

Lawrenceville, NJ
10,001-50,000 employees

treatment of follicular lymphoma

Disease Area: Rare Disease
Source
Source
February 12, 2026

NorthSea Therapeutics B.V.

Orphan Drug Designation

treatment of intestinal failure associated liver disease

Disease Area: Rare Disease
Source
Source
February 12, 2026

FBD Biologics Limited

Orphan Drug Designation

treatment of gastric cancer

Disease Area: Rare Disease
Source
Source
February 11, 2026

MeiraGTx UK II Ltd

Orphan Drug Designation

treatment of inherited retinal dystrophy due to GUCY2D gene mutations

Disease Area: Rare Disease
Source
Source
February 11, 2026

Xadcera Biopharmaceutical (Suzhou) Co., Ltd.

Orphan Drug Designation

treatment of pancreatic cancer

Disease Area: Rare Disease
Source
Source
February 10, 2026

2A Biosciences, Inc.

Orphan Drug Designation

treatment of neurotrophic keratopathy

Disease Area: Rare Disease
Source
Source
February 9, 2026

Hangzhou Zhongmei Huadong Pharmaceutical Co., Ltd

Orphan Drug Designation

treatment of gastric cancer, including gastroesophageal junction cancer

Disease Area: Rare Disease
Source
Source
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