VivoSeq
Other

Smith-Lemli-Opitz Syndrome

6

Pipeline Programs↗

1

Active Trials↗

1

Companies Involved↗

Smith-Lemli-Opitz syndrome is an early-stage landscape with 6 programs across 1 company. The most active players are Mirum Pharmaceuticals, Inc.. 1 therapy is approved.

Intelligence

Pipeline Metrics

Competitive Density

30/100

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.

M

Mirum Pharmaceuticals, Inc.

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Trial Status

5

clinical trials

Completed4 · 80%
Recruiting1 · 20%

Clinical Trials

Trial Activity

Simvastatin Therapy in Smith-Lemli-Opitz Syndrome
Completed·—·Phase 2·NCT00064792·23 enrolled
Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)
Recruiting·UUniversity of Colorado, Denver·Phase 2·NCT01773278·100 enrolled
Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome
Completed·—·Phase 1/2·NCT00272844·23 enrolled
Smith-Lemli-Opitz Syndrome and Cholic Acid
Completed·UUniversity of Nebraska·Phase 1/2·NCT03720990·12 enrolled
Clinical and Basic Investigations Into Smith-Lemli-Opitz Syndrome
Completed·Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)·N/A·NCT00001721·130 enrolled

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