VivoSeq
Other

Medium-Chain Acyl-Coa Dehydrogenase Deficiency

2

Pipeline Programs↗

0

Active Trials↗

2

Companies Involved↗

Medium-chain Acyl-CoA Dehydrogenase Deficiency is an early-stage landscape with 2 programs across 2 companies. The most active players are Jerry Vockley, MD, PhD, Ultragenyx.

Intelligence

Pipeline Metrics

Competitive Density

16/100

Pipeline Velocity

11/100

Mech. Concentration

N/A

Completion Rate

0/100

Attrition Rate

67/100

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.

J

Jerry Vockley, MD, PhD

1 program

Phase 2Ultragenyx logo

Ultragenyx

1 program

Phase 2

Pipeline

Programs by Phase

Preclinical
No programs
Phase 1
No programs
Phase 22

Triheptanoin

Ultragenyx

Sodium Phenylbutyrate

Jerry Vockley, MD, PhD

Phase 3
No programs
Approved
No programs

Analysis

Trial Status

3

clinical trials

Withdrawn2 · 67%
Suspended1 · 33%

Clinical Trials

Trial Activity

A Phase 2, Open-Label, Fixed Dose Study to Evaluate the Use of Sodium Phenylbutyrate (ACER-001) in the Treatment of Pediatric and Adult Patients With MCAD Deficiency Caused by the Common ACADM c.985 A>G (K304E) Mutation
Suspended·JJerry Vockley, MD, PhD·Phase 2·NCT06069375·24 enrolled
A Phase II, Escalating Dose, Open Label Study to Evaluate the Safety of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Withdrawn·JJerry Vockley, MD, PhD·Phase 2·NCT06067802
A Phase II, Open Label Study to Evaluate the Safety of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Withdrawn·JJerry Vockley, MD, PhD·Phase 2·NCT07097311

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