Skip to main content

Methylmalonic Acidemia

1
Pipeline Programs
4
Companies
7
Clinical Trials
2 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
2100%
+ 5 programs with unclassified modality

Competitive Landscape

4 companies ranked by most advanced pipeline stage

AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
2 programs
1
hLB-001Phase 1/21 trial
hLB-001N/A1 trial
Active Trials
NCT05506254Active Not Recruiting4Est. Jan 2037
NCT04581785Terminated4Est. Jan 2023
Moderna
ModernaCAMBRIDGE, MA
3 programs
"The MaP Study": Mapping the Patient Journey in MMA and PAN/A1 trial
mRNA-3705PHASE_1_2RNA Therapeutic1 trial
mRNA-3705PHASE_1_2RNA Therapeutic1 trial
Active Trials
NCT03484767Completed97Est. May 2021
NCT04899310Active Not Recruiting74Est. Jul 2028
NCT05295433Recruiting56Est. Apr 2034
Recordati
RecordatiFrance - Saint-Victor
1 program
Carglumic AcidN/A1 trial
Active Trials
NCT04176523Recruiting95Est. Jul 2029
Genome & Company
Genome & CompanyKorea - Suwon
1 program
RegistryN/A1 trial
Active Trials
NCT05438485Terminated11Est. Oct 2023

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
ModernamRNA-3705
ModernamRNA-3705
AstraZenecahLB-001
AstraZenecahLB-001
Genome & CompanyRegistry
RecordatiCarglumic Acid
Moderna"The MaP Study": Mapping the Patient Journey in MMA and PA

Clinical Trials (7)

Total enrollment: 341 patients across 7 trials

NCT05295433ModernamRNA-3705

An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705

Start: Mar 2022Est. completion: Apr 203456 patients
Phase 1/2Recruiting
NCT04899310ModernamRNA-3705

A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

Start: Aug 2021Est. completion: Jul 202874 patients
Phase 1/2Active Not Recruiting
NCT04581785AstraZenecahLB-001

Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia

Start: May 2021Est. completion: Jan 20234 patients
Phase 1/2Terminated
NCT05506254AstraZenecahLB-001

Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy

Start: Jul 2022Est. completion: Jan 20374 patients
N/AActive Not Recruiting
NCT05438485Genome & CompanyRegistry

Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia

Start: Aug 2021Est. completion: Oct 202311 patients
N/ATerminated
NCT04176523RecordatiCarglumic Acid

Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach

Start: Jan 2019Est. completion: Jul 202995 patients
N/ARecruiting
NCT03484767Moderna"The MaP Study": Mapping the Patient Journey in MMA and PA

"The MaP Study": Mapping the Patient Journey in MMA and PA

Start: Mar 2018Est. completion: May 202197 patients
N/ACompleted

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

2 actively recruiting trials targeting 341 patients
4 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.