Methodology

All entries for: Rare Disease

January 29, 2026

Parabilis Medicines, Inc.

Orphan Drug Designation

treatment of desmoid tumors

Disease Area: Rare Disease

Source

January 29, 2026

AbbVie Inc.

Orphan Drug Designation

treatment of pancreatic cancer

Disease Area: Rare Disease

Source

January 29, 2026

Partner Therapeutics, Inc.

Orphan Drug Designation

treatment of cholangiocarcinoma

Disease Area: Rare Disease

Source

January 26, 2026

Insmed Incorporated

Orphan Drug Designation

treatment of pulmonary arterial hypertension

Disease Area: Rare Disease

Source

January 26, 2026

TB Alliance

Orphan Drug Designation

treatment of tuberculosis

Disease Area: Rare Disease

Source

January 26, 2026

Lyell Immunopharma, Inc.

Orphan Drug Designation

South San Francisco, California

201-500 employees

treatment of diffuse large B-cell lymphoma (DLBCL)/high grade B-cell lymphoma (HGBCL) with MYC and BCL2 rearrangements

Disease Area: Rare Disease

Source

January 23, 2026

Cy Biopharma AG

Orphan Drug Designation

treatment of complex regional pain syndrome

Disease Area: Rare Disease

Source

January 23, 2026

Beijing Jitai Life Sciences Ltd.

Orphan Drug Designation

treatment of squamous non-small cell lung cancer

Disease Area: Rare Disease

Source

January 23, 2026

Shanghai Vitalgen BioPharma Co., Ltd.

Orphan Drug Designation

treatment of aromatic L-amino acid decarboxylase (AADC) deficiency

Disease Area: Rare Disease

Source

January 22, 2026

Vertex Pharmaceuticals Incorporated

Orphan Drug Designation

Treatment of membranous nephropathy

Disease Area: Rare Disease

Source