Methodology

All entries for: Orphan Drug Designation

January 15, 2026

Revio Therapeutics LLP

Orphan Drug Designation

treatment of Duchenne muscular dystrophy

Disease Area: Rare Disease
Source
Source
January 13, 2026

Shanghai Vitalgen BioPharma Co., Ltd.

Orphan Drug Designation

treatment of Gaucher disease

Disease Area: Rare Disease
Source
January 13, 2026

University of California, San Francisco

Orphan Drug Designation

treatment of acute myeloid leukemia

Disease Area: Rare Disease
Source
Source
January 13, 2026

HemaNiche Biotech Co., Ltd

Orphan Drug Designation

treatment of post-hematopoietic stem cell transplantation thrombocytopenia

Disease Area: Rare Disease
Source
Source
January 13, 2026

Heronova Pharmaceuticals, LLC

Orphan Drug Designation

treatment of Stargardt disease

Disease Area: Rare Disease
Source
Source
January 13, 2026

Biotle Biotechnology (Hangzhou) Co., Ltd.

Orphan Drug Designation

treatment of multiple myeloma

Disease Area: Rare Disease
Source
Source
January 13, 2026

Galderma Research and Development, LLC

Orphan Drug Designation

treatment of systemic sclerosis

Disease Area: Rare Disease
Source
Source
January 13, 2026

BPGbio Inc.

Orphan Drug Designation

treatment of primary CoQ10 deficiency

Disease Area: Rare Disease
Source
Source
January 5, 2026

Delnaggar Biotech Group

Orphan Drug Designation

FDA orphan drug designation: treatment of high-grade anal intraepithelial neoplasia (HGAIN)

Disease Area: Rare Diseases
Source
Source
January 5, 2026

Nanjing RegeneCore Biotech Co.

Orphan Drug Designation

FDA orphan drug designation: treatment of acute myeloid leukemia

Disease Area: Rare Diseases
Source
Source
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