/
Rare Disease
34Diseases↗
909Pipeline Programs↗
328Active Trials↗
Momentum Score:100/100
Landscape
Disease Coverage Map
Bubble size = actively recruiting trials · Click to explore
Analysis
Phase Distribution
Analysis
Trial Status
1,636
clinical trials
Completed967 · 59%
Terminated167 · 10%
Recruiting165 · 10%
Active, not recruiting104 · 6%
Unknown status84 · 5%
Withdrawn56 · 3%
Other93 · 6%
Competitive
Top Companies Companies ranked by number of pipeline programs they run in this therapeutic area.
38programs
32programs
32programs
29programs
29programs
Landscape
Diseases
SMA
Most programs0
Programs
1,638
Trials
4
Recruiting
Cystic Fibrosis
Most trials342
Programs
40
Trials
31
Recruiting
Pre
63
P1
73
P2
147
P3
55
Appr
54
Most programs:
Vertex Pharmaceuticals
Vertex PharmaceuticalsDuchenne Muscular Dystrophy
Highest activity110
Programs
28
Trials
16
Recruiting
Pre
16
P1
21
P2
57
P3
32
Appr
10
Most programs:
Sarepta Therapeutics
Sarepta TherapeuticsHemophilia a
270 programs54 trialsApproved
Paroxysmal Nocturnal Hemoglobinuria
137 programs28 trialsPhase 3
Spinal Muscular Atrophy
35 programs28 trialsApproved
Hereditary Angioedema
38 programs16 trialsApproved
Fabry Disease
229 programs19 trialsApproved
Pompe Disease
135 programs9 trialsApproved
Fanconi Anemia
234 programs8 trialsApproved
Gaucher Disease
31 programs7 trialsApproved
Acromegaly
331 programs6 trialsApproved
Phenylketonuria
121 programs12 trialsApproved
Hereditary Hemorrhagic Telangiectasia
23 programs7 trialsApproved
Hypothalamic Obesity
14 programs4 trialsApproved
Familial Mediterranean Fever
19 programs4 trialsApproved
Von Hippel-Lindau Disease
111 programs1 trialPhase 3
Cryopyrin-Associated Periodic Syndrome
9 programs1 trialPhase 3
Hereditary Transthyretin Amyloidosis
7 programs1 trialApproved
Inborn Error of Immunity
7 programs0 trialsApproved
Schnitzler Syndrome
4 programs0 trialsPhase 2
Muckle-Wells Syndrome
4 programs0 trialsPhase 3
Blau Syndrome
2 programs2 trialsApproved
Batten Disease
3 programs0 trialsPhase 2
SAPHO Syndrome
1 program1 trialPhase 2
Hereditary Hemorrhagic Telangiectasia (HHT)
10 programs2 trialsPhase 1
Erdheim-Chester Disease
1 program0 trialsPhase 2
Mevalonate Kinase Deficiency
1 program0 trialsPreclinical
Von Hippel Lindau
0 programs1 trial
Inborn Errors of Metabolism
10 programs0 trials
Showing top 30 diseases by activity. 4 more diseases in this therapeutic area.Activity = a momentum score from recent trial starts, number of sponsoring companies, and total trial volume.
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)