Methodology

All entries for: Orphan Drug Designation

January 20, 2026

University of Granada

Orphan Drug Designation

treatment of primary CoQ10 deficiency

Disease Area: Rare Disease

Source

January 16, 2026

Gleeson Lab - UCSD

Orphan Drug Designation

treatment of primary CoQ10 deficiency

Disease Area: Rare Disease

Source

January 15, 2026

XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd

Orphan Drug Designation

treatment of malignant glioma

Disease Area: Rare Disease

Source

January 15, 2026

Advaccine Biopharmaceuticals Suzhou Co., Ltd.

Orphan Drug Designation

treatment of recurrent respiratory papillomatosis

Disease Area: Rare Disease

Source

January 15, 2026

Daiichi Sankyo, Inc.

Orphan Drug Designation

treatment of esophageal cancer

Disease Area: Rare Disease

Source

January 15, 2026

NexEos Diagnostics Inc. (dba NexEos Bio)

Orphan Drug Designation

treatment of vernal keratoconjunctivitis

Disease Area: Rare Disease

Source

January 15, 2026

Nanjing Reju Therapeutics Co., Ltd.

Orphan Drug Designation

treatment of idiopathic pulmonary fibrosis

Disease Area: Rare Disease

Source

January 15, 2026

Ascentage Pharma Group, Inc.

Orphan Drug Designation

treatment of myelodysplastic syndrome

Disease Area: Rare Disease

Source

January 15, 2026

Lantern Pharma Inc.

Orphan Drug Designation

treatment of soft tissue sarcoma

Disease Area: Rare Disease

Source

January 15, 2026

Atossa Therapeutics, Inc.

Orphan Drug Designation

treatment of Duchenne muscular dystrophy

Disease Area: Rare Disease

Source