VivoSeq
Neurology

Niemann-Pick Disease Type C

6

Pipeline Programs↗

2

Active Trials↗

3

Companies Involved↗

Niemann-Pick disease type C is an early-stage landscape with 6 programs across 3 companies. The most active players are Cyclo Therapeutics, Inc., Arbor Pharmaceuticals, Inc., Solvay Pharmaceuticals. 2 therapies are approved with 3 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

31/100

Pipeline Velocity

0/100

Mech. Concentration

100/100

Completion Rate

100/100

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.

C

Cyclo Therapeutics, Inc.

2 programs

Phase 3A

Arbor Pharmaceuticals, Inc.

1 program

ApprovedS

Solvay Pharmaceuticals

1 program

Approved

Pipeline

Programs by Phase

Analysis

Trial Status

4

clinical trials

Recruiting1 · 25%
Completed1 · 25%
Unknown status1 · 25%
Active, not recruiting1 · 25%

Clinical Trials

Trial Activity

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
Recruiting·IIntraBio Inc·Phase 3·NCT05163288·53 enrolled
Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
Completed·ZZevraDenmark·Phase 2/3·NCT02612129·50 enrolled
Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C
Unknown status·CCyclo Therapeutics, Inc.·Phase 1·NCT03893071·12 enrolled
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
Active, not recruiting·CCyclo Therapeutics, Inc.·Phase 3·NCT04860960·94 enrolled

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