Methylmalonic Acidemia
2
Pipeline Programs
2
Companies
4
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
2
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
RNA Therapeutic
2100%
+ 2 programs with unclassified modality
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
ModernamRNA-3705
ModernamRNA-3705
RecordatiCarglumic Acid
Moderna"The MaP Study": Mapping the Patient Journey in MMA and PA
Clinical Trials (4)
Total enrollment: 322 patients across 4 trials
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
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
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Start: Jan 2019Est. completion: Jul 202995 patients
N/ARecruiting
"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 322 patients
2 companies competing in this space