VivoSeq
Other

Familial Dysautonomia

6

Pipeline Programs↗

2

Active Trials↗

4

Companies Involved↗

Familial dysautonomia is an early-stage landscape with 6 programs across 4 companies. The most active players are McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc., Alnylam Pharmaceuticals, Boehringer Ingelheim. 1 therapy is approved with 3 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

31/100

Pipeline Velocity

0/100

Mech. Concentration

20/100

Completion Rate

67/100

Attrition Rate

20/100

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.

M

McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.

1 program

Phase 2Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals

1 program

Phase 2Boehringer Ingelheim logo

Boehringer Ingelheim

1 program

Phase 3G

Glaxo Dba Glaxosmithkline

1 program

Approved

Pipeline

Programs by Phase

Analysis

Trial Status

5

clinical trials

Completed2 · 40%
Withdrawn1 · 20%
Enrolling by invitation1 · 20%
Active, not recruiting1 · 20%

Clinical Trials

Trial Activity

The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
Withdrawn·NNYU Langone Health·Phase 2·NCT02608931
The Effects Of Bronchodilator Therapy On Respiratory And Autonomic Function In Patients With Familial Dysautonomia
Completed·NNYU Langone Health·Phase 3·NCT01987219·15 enrolled
Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Enrolling by invitation·NNYU Langone Health·Phase 2·NCT06148311·15 enrolled
Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Active, not recruiting·NNYU Langone Health·Phase 2·NCT06128356·5 enrolled
Carbidopa for the Treatment of Nausea and Vomiting in Familial Dysautonomia
Completed·NNYU Langone Health·Phase 3·NCT01212484·12 enrolled

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