/
Rare Disease
34Diseases↗
909Pipeline Programs↗
308Active Trials↗
Momentum Score:100/100
Landscape
Disease Coverage Map
Bubble size = actively recruiting trials · Click to explore
Analysis
Phase Distribution
Analysis
Trial Status
1,597
clinical trials
Completed958 · 60%
Recruiting165 · 10%
Terminated160 · 10%
Active, not recruiting99 · 6%
Unknown status84 · 5%
Withdrawn56 · 4%
Other75 · 5%
Competitive
Top Companies Companies ranked by number of pipeline programs they run in this therapeutic area.
37programs
31programs
30programs
29programs
28programs
Landscape
Diseases
SMA
Most programs0
Programs
1,638
Trials
5
Recruiting
Cystic Fibrosis
Most trials342
Programs
40
Trials
30
Recruiting
Pre
64
P1
69
P2
146
P3
54
Appr
54
Most programs:
Vertex Pharmaceuticals
Vertex PharmaceuticalsDuchenne Muscular Dystrophy
Highest activity110
Programs
28
Trials
17
Recruiting
Pre
14
P1
21
P2
57
P3
29
Appr
5
Most programs:
Sarepta Therapeutics
Sarepta TherapeuticsHemophilia a
470 programs54 trialsApproved
Paroxysmal Nocturnal Hemoglobinuria
337 programs28 trialsPhase 3
Spinal Muscular Atrophy
235 programs28 trialsApproved
Hereditary Angioedema
38 programs16 trialsApproved
Fabry Disease
229 programs19 trialsApproved
Pompe Disease
35 programs9 trialsApproved
Fanconi Anemia
134 programs8 trialsApproved
Gaucher Disease
231 programs7 trialsApproved
Acromegaly
131 programs6 trialsApproved
Phenylketonuria
321 programs12 trialsApproved
Hereditary Hemorrhagic Telangiectasia
123 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)