Methodology

All entries for: Rare Disease

February 5, 2026

ENCell Co., Ltd.

Orphan Drug Designation

treatment of Duchenne muscular dystrophy

Disease Area: Rare Disease
Source
Source
February 5, 2026

Hellene Solomon Wan, MD - Induvidual sponsor.

Orphan Drug Designation

treatment of neonatal diabetes mellitus (monogenic forms of diabetes mellitus that present in infancy)

Disease Area: Rare Disease
Source
Source
February 4, 2026

Zydus Healthcare (USA), LLC,

Orphan Drug Designation

Treatment of Sickle Cell Disease

Disease Area: Rare Disease
Source
Source
February 4, 2026

Novartis Pharmaceuticals Corporation

Orphan Drug Designation

treatment of progressive supranuclear palsy

Disease Area: Rare Disease
Source
Source
February 3, 2026

Immatics US, Inc

Orphan Drug Designation

treatment of Stage II and higher cutaneous melanoma (CM), in human leukocyte antigen (HLA)-A*02:01-positive patients

Disease Area: Rare Disease
Source
Source
February 3, 2026

LBR Regulatory and Clinical Consulting Services, Inc.

Orphan Drug Designation

treatment of tuberous sclerosis complex

Disease Area: Rare Disease
Source
Source
February 2, 2026

ODDIFACT SAS

Orphan Drug Designation

treatment of neuromyelitis optica spectrum disorder

Disease Area: Rare Disease
Source
Source
February 2, 2026

Solve FSHD

Orphan Drug Designation

treatment of facioscapulohumeral muscular dystrophy

Disease Area: Rare Disease
Source
Source
February 2, 2026

Vertex Pharmaceuticals Incorporated

Orphan Drug Designation

treatment of myasthenia gravis

Disease Area: Rare Disease
Source
Source
February 2, 2026

AstraZeneca Pharmaceuticals LP

Orphan Drug Designation

treatment of malignant glioma

Disease Area: Rare Disease
Source
Source
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