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Rare Disease
34Diseases↗
299Pipeline Programs↗
351Active Trials↗
Momentum Score:100/100
Landscape
Disease Coverage Map
Bubble size = actively recruiting trials · Click to explore
Analysis
Phase Distribution
Analysis
Trial Status
1,676
clinical trials
Completed982 · 59%
Recruiting179 · 11%
Terminated170 · 10%
Active, not recruiting114 · 7%
Unknown status84 · 5%
Withdrawn56 · 3%
Other91 · 5%
Competitive
Top Companies Companies ranked by number of pipeline programs they run in this therapeutic area.
Landscape
Diseases
SMA
Most programs2
Programs
2,421
Trials
3
Recruiting
Pre
—
P1
2
P2
—
P3
—
Appr
—
Most programs:
AbbVie
AbbVieCystic Fibrosis
Most trials84
Programs
75
Trials
36
Recruiting
Pre
45
P1
4
P2
18
P3
10
Appr
30
Most programs:
Vertex Pharmaceuticals
Vertex PharmaceuticalsHemophilia a
Highest activity39
Programs
68
Trials
27
Recruiting
Pre
17
P1
3
P2
2
P3
9
Appr
3
Most programs:
Roche
RocheDuchenne Muscular Dystrophy
441 programs51 trialsApproved
Paroxysmal Nocturnal Hemoglobinuria
20 programs37 trialsApproved
Spinal Muscular Atrophy
14 programs40 trialsApproved
Fabry Disease
112 programs28 trialsPhase 3
Hereditary Angioedema
113 programs19 trialsApproved
Phenylketonuria
110 programs18 trialsApproved
Pompe Disease
7 programs19 trialsApproved
Fanconi Anemia
12 programs11 trialsApproved
Gaucher Disease
7 programs16 trialsApproved
Hereditary Hemorrhagic Telangiectasia
7 programs12 trialsApproved
Acromegaly
9 programs6 trialsApproved
Hypothalamic Obesity
14 programs5 trialsApproved
Familial Mediterranean Fever
22 programs6 trialsApproved
Hereditary Hemorrhagic Telangiectasia (HHT)
22 programs5 trialsPhase 1
Von Hippel-Lindau Disease
13 programs2 trialsPhase 3
Blau Syndrome
2 programs2 trialsPhase 3
Inborn Error of Immunity
2 programs0 trialsApproved
Cryopyrin-Associated Periodic Syndrome
2 programs0 trialsPreclinical
Batten Disease
2 programs0 trialsPhase 2
Hereditary Transthyretin Amyloidosis
1 program1 trialApproved
SAPHO Syndrome
1 program1 trialPhase 3
Von Hippel Lindau
0 programs2 trials
Muckle-Wells Syndrome
1 program0 trialsPreclinical
Erdheim-Chester Disease
0 programs1 trial
Inborn Errors of Metabolism
10 programs1 trial
VHL - Von Hippel-Lindau Syndrome
0 programs0 trials
Schnitzler Syndrome
0 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)