VivoSeq
Rare Disease

Blau Syndrome

2

Pipeline Programs↗

2

Active Trials↗

1

Companies Involved↗

Blau syndrome is an early-stage landscape with 2 programs across 1 company. The most active players are Prism Pharmaceuticals. 1 therapy is approved.

Intelligence

Pipeline Metrics

Competitive Density

21/100

Pipeline Velocity

0/100

Mech. Concentration

100/100

Completion Rate

N/A

Attrition Rate

0/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

P

Prism Pharmaceuticals

1 program

Preclinical

Pipeline

Programs by Phase

Preclinical1

Tofacitinib

Prism Pharmaceuticals

Phase 1
No programs
Phase 2
No programs
Phase 3
No programs
Approved1

Janus Kinase Inhibitor

Peking Union Medical College Hospital

Analysis

Trial Status

2

clinical trials

Enrolling by invitation2 · 100%

Clinical Trials

Trial Activity

Efficacy and Safety of Tofacitinib in Refractory Blau Syndrome
Enrolling by invitation·PPeking Union Medical College Hospital·Phase 4·NCT06660329·30 enrolled
Effective Treatment of Jak1/3 Inhibitor in Blau Syndrome
Enrolling by invitation·TTongji Hospital·N/A·NCT06688838·24 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)